Rare Diseases 2013 PDF

2013 REPORT
Medicines in Development
Rare Diseases
A Report on Orphan Drugs in the Pipeline
presented by america’s biopharmaceutical research companies
More Than 450 Medicines in Development for

Rare Diseases
Rare diseases, when taken together, are A major area of this research targets
Orphan Drugs in Development*
not that rare at all. In fact, according to rare cancers, accounting for more than
Application the National Institutes of Health (NIH), one-third of all rare disease medicines in
Submitted
30 million Americans have one of the development. Other top research areas
Phase III
nearly 7,000 diseases that are officially include genetic disorders, neurologi-
Phase II deemed “rare” because alone they each cal conditions, infectious diseases and
Phase I affect fewer than 200,000 people in autoimmune disorders.
the United States. Sometimes, only
Despite some recent victories, research
a few hundred patients are known to
105 into treatments for rare diseases is a
have a particular rare disease.
daunting quest. This ongoing innovation
Simply receiving a diagnosis of a rare and the hundreds of new medicines in
disease often becomes a frustrating development now offer hope that physi-
85 quest, since many doctors may have nev- cians will have new treatment options
er before heard of or seen the disease. for patients confronting a rare disease.
This is, however, a time of great

progress and hope. Biopharmaceutical Contents 
65
research is entering an exciting new era
Innovative Orphan Drugs
with a growing understanding of the
in the Pipeline.......................................... 2
human genome. Scientific advances
have given researchers new tools to Orphan Drug Approvals............................4
explore rare diseases, which are often Challenges in Clinical Trials.......................6
more complex than common diseases. Prescription Drug User Fee Act
32
(PDUFA).................................................. 7
28 In 2012 alone, 13 orphan drugs were
approved for rare diseases, including Rare Disease Facts and Statistics............... 7
20 therapies for Cushing disease, cystic Orphan Drugs in Development..................8
fibrosis and Gaucher disease. America’s Glossary................................................ 52
biopharmaceutical research companies Drug Development/
are continuing that progress with 452
Approval Process...................................56
medicines and vaccines in development
so tic
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Inf ders
Ne isea us
for rare diseases. The medicines listed

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in this report are either in clinical trials

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nc
or under review by the Food and Drug

Ca
*Some medicines are listed in more than one category. Administration (FDA).
Key Issues
Innovative Orphan Drugs in the function and, as dystrophin expression increases, there have
been demonstrated improvements in patients’ ability to walk.
Pipeline for Rare Diseases
Potential New Treatment for a Genetic Disease in Infants
The 452 medicines and vaccines in development for rare dis- —Hypophosphatasia is a rare inherited bone disease that
eases employ exciting new scientific and technical knowledge. results from a genetic mutation which hinders the forma-
Many of the medicines, which offer hope for those suffering tion of bones and teeth and can result in substantial skeletal
from one of the nearly 7,000 rare diseases, represent innova- abnormalities. Severely affected infants often have persistent
tive new ways to target disease, including: bone disease or die from respiratory insufficiency due to
Targeted RNAi Therapy Approach for Duchenne Muscular progressive chest deformity from poorly developed bones.
Dystrophy—In clinical trials, RNAi therapies have shown po- Currently, there are no approved medicines for this disease.
tential in treating neuromuscular disorders such as Duchenne One therapy in development delivers the enzyme necessary
muscular dystrophy (DMD), which affects about 1 in every for proper bone growth that patients with hypophosphatasia
3,500 to 6,000 male births each year in the United States. are missing.
In DMD, DNA deletions cause mutations in important genes Treatments for Patients with Debilitating Lung Disease—
that encode for dystrophin, a structural protein found in Idiopathic pulmonary fibrosis (IPF) is a debilitating and almost
normal muscle. The loss of this protein causes muscle fibers uniformly fatal disease in which patients experience progres-
to disintegrate faster than they can be regenerated. One sive difficulty breathing due to scarring of the lungs. There
medicine in development targets restoration of dystrophin are currently no effective treatment options available, and
Medicines in Development By Disease and Phase

Some medicines are listed in more than one category.
Autoimmune Disorders 18 Application

Submitted
Blood Disorders 7
Phase III
Cancer 105
Phase II
Cancer, Blood 65
Cancer-Related Phase I
10
Cancer, Skin 15
Cardiovascular Diseases 4
Digestive Disorders 14
Eye Disorders 16
Genetic Disorders 85
Growth Disorders 7
Infectious Diseases 28
Neurological Disorders 32
Respiratory Disorders 20
Skin Conditions 2
Transplantation 14
Other 35
2 Medicines in Development Rare Diseases 2013

Key Issues
ALS: Fighting a Devastating Disease—Amyotrophic lateral
RARE DISEASES BIG IMPACT sclerosis (ALS), or Lou Gehrig’s disease, is a progressive
neurodegenerative disease that causes the brain to lose
control over body movement, ultimately resulting in paralysis
and death. The one drug approved to treat ALS can mod-
estly slow progression of the disease, but new treatments
are needed. As our scientific understanding of the disease
has grown, researchers are pursuing many new approaches
to halt or slow progression, including the use of the patient’s
own bone marrow stem-cells to create healthy neuron-like
RAR
7,000 cells to replace diseased neurons. Other trials are studying
ways to prompt the immune system to protect neurons af-
fected by ALS.
E D IS E
ASES WORLDWIDE Two Targets to Fight Leukemia—A potential first-in-class
medicine for acute lymphoblastic leukemia (ALL) is a bispe-
cific T-cell engager antibody designed to focus the body’s
cell destroying T-cells against cells expressing CD19, a
Source: National Institutes of Health protein found on the surface of B-cell-derived leukemia and
lymphoma. The modified antibodies are designed to engage
two different targets simultaneously, linking the T-cells to
the average patient with IPF dies within three years of diag-
cancer cells.
nosis. A medicine in development targets connective tissue
growth factor, which is elevated in the lungs of IPF patients. Combination Vaccine Treatment for Pancreatic Cancer—A
Researchers recently announced promising results from a potential treatment for pancreatic cancer is a combination of
Phase II trial in which 60 percent of IPF patients were able two therapeutic vaccines. The treatment combines a Listeria-
to stabilize their disease or experience improvement in lung based vaccine that has been engineered to express the
function.
Orphan Drug Act of 1983— RARE DISEASES BIG IMPACT

A Success Story
Recognizing the scarcity of medicines to treat rare
diseases with very small patient populations, the
Orphan Drug Act of 1983 provided incentives to
companies developing rare disease treatments. BY DEFINITION, A RARE DISEASE AFFECTS
<200,000
Over the last 30 years, more than 400 medicines
representing 447 separate indications have been
approved to treat rare diseases, compared to
fewer than 10 in the 1970s. As of September
15, 2013, the FDA has granted the orphan drug
patients in the United States
designation to 2,899 potential therapies.
Source: National Institutes of Health
Medicines in Development Rare Diseases 2013 3

Key Issues
tumor-associated antigen mesothelin and allogeneic pancre-

atic cancer cells that are genetically-modified to secrete the
immune-stimulant, granulocyte-macrophage colony stimu-
RARE DISEASES BIG IMPACT
lating factor (GM-CSF). The cells are irradiated to prevent
further cell growth although they stay metabolically active.
Sequential administration of the vaccines in animal studies
have demonstrated enhanced tumor-specific T-cell and anti-
tumor responses.
IN TOTAL, RARE DISEASE IMPACT
Orphan Drug Approvals for

Rare Diseases
30 MILLION
Since passage of the Orphan Drug Act in 1983, more than
400 medicines for rare diseases have been brought to mar-
ket. Below are some key approvals for rare diseases in recent
years that represent new technologies or a more defined 1 IN 10 AMERICANS
patient population target.
Source: National Institutes of Health
Zelboraf® (vemurafenib), a personalized medicine, was
approved for the treatment of unresectable or metastatic
melanoma that expresses a gene mutation called BRAF protein is involved in regulating cell growth but a mutated
V600E. It was approved with a first-of-its-kind companion form is found in about half of the late-stage melanoma
diagnostic (4800 BRAF V600 Mutation Test) to help de- cases. Zelboraf is able to block the function of the V600E-
termine if a patient has the gene mutation. A normal BRAF mutated BRAF protein.
Seeking New Treatments for Children with a Rare Disease
The National Institutes of Health estimates that 50 percent of people affected by rare diseases are children,
making rare diseases a particularly deadly and debilitating concern for children worldwide. Rare diseases
are responsible for 35 percent of deaths in the first year of life and 30 percent of children with a rare disease
will not live to see their fifth birthday.
Encouragingly, one in three of the nearly 3,000 treatments with orphan designation are for children. In
addition to the Orphan Drug Act, two other laws have made a significant impact on pediatric research. The
Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) have resulted
in a wealth of useful information about dosing, safety, and efficacy. According to the FDA, BPCA and PREA
have resulted in 467 pediatric labeling changes since 1988. Together, BPCA and PREA have helped foster
research and greatly advanced our ability to treat pediatric patients.
BPCA and PREA were permanently reauthorized by the Food and Drug Administration Safety and Innovation
Act of 2012 (FDASIA). By providing a predictable regulatory environment, the permanent reauthorization
will help ensure that pediatric research by biopharmaceutical companies continues to advance children’s
medical care. FDASIA also required the FDA to hold a public meeting and issue a report on encouraging and
accelerating development of new therapies for pediatric rare diseases.

Key Issues
cally, Ilaris was approved to treat two types of CAPS: familial

cold-auto-inflammatory syndrome (FACS) and Muckle-Wells
“In the last five years, one-third of all new
syndrome (MWS). CAPS is a serious auto-inflammatory
drug approvals were for rare diseases” disease that lasts a lifetime with symptoms such as fever,
—FDA headache, fatigue, skin rash, painful joints and muscles.
CAPS is caused by a single gene mutation that leads to
overproduction of interleukin-1 beta, which causes sustained
Adcetris® (brentuximab vedotin), the first in a new class of inflammation and tissue damage. Ilaris rapidly and selectively
antibody-drug conjugates (ADCs), was approved to treat blocks production of interleukin-1 beta.
Hodgkin lymphoma and systemic anaplastic large cell lym-
phoma (ALCL), a rare type of lymphoma that represents only Kalydeco™ (ivacaftor) was the first medicine approved to
3 percent of all non-Hodgkin lymphomas. ADCs combine treat the underlying cause of cystic fibrosis (CF) and not just
a monoclonal antibody and a therapeutic drug, where the the symptoms of the disease. It targets a defective protein
antibody directs the therapeutic to target the cancerous cells. to help achieve sustained improvement in lung function.
It is also the first FDA-approved drug for Hodgkin lymphoma Kalydeco was approved for use in people with cystic fibrosis
in more than 30 years and the first to specifically treat ALCL. (ages 6 and older) who have at least one copy of the G551D
Adcetris is composed of an anti-CD30 monoclonal antibody mutation in the CF transmembrane conductance regula-
and a microtubule disrupting agent, allowing it to release its tor (CFTR) gene. The defect affects a small portion of CF
therapeutic drug once inside the CD30-expressing tumor cells. patients—about 5 percent or 1,200 of the 30,000 CF suf-
ferers—but it also provides hope that knowledge gained will
Ilaris® (canakinumab), a fully human monoclonal antibody, lead to treatments that will help even more CF patients.
was approved to treat adults and children as young as age 4
with cryopyrin-associated periodic syndrome (CAPS). Specifi-
RARE DISEASES BIG IMPACT Patient Engagement

The biopharmaceutical research industry is
committed to close collaboration and regular
coordination with rare disease patient groups
DRU
AN SINCE 1983, NEARLY and other rare disease stakeholders. We must
2,900
work together towards building a positive policy
GA
ORPH
environment to help us meet the public health

challenges that rare diseases present.
CT
ORPHAN DRUG
1983 DESIGNATIONS
RESEARCH PROGRESS HOPE

Source: Office of Orphan Products Development,
Food and Drug Administration

Key Issues
found genes associated with diseases such as myotonic dys-
RARE DISEASES BIG IMPACT trophy, ALS, cystic fibrosis, progeria and neurofibromatosis
types 1 and 2. These breakthroughs are a crucial step toward
new treatments.
FDA 400 APPROVED

TREATMENTS Evolving genetic research may offer a solution to the chal-
lenges of clinical development. Genetic markers may make it
possible to identify a patient population in advance and allow
clinical trials with a smaller number of participants.
Before a potential new treatment can be approved, it must

be tested in clinical trials. It is often difficult to find patients
to volunteer in clinical trials, and rare diseases pose an even
A GOOD START, but greater challenge. Specific rare disease patient populations
are very small, geographically dispersed and often include
<5%
Source: Food and Drug Administration,
of RARE DISEASES
HAVE A TREATMENT
children. The biopharmaceutical sector is working with pa-
tient advocacy organizations to identify and advance better
ways to connect patients to biopharmaceutical and academic
researchers conducting clinical trials.
EveryLife Foundation for Rare Diseases
Physicians and patients can find out about clinical trials being
conducted all over the country in collaboration with local
Challenges in Clinical Trials for institutions by accessing www.clinicaltrials.gov, a database
Rare Diseases sponsored by the National Institutes of Health. Information
on clinical trials and medicines in development is also avail-
Advances in science and technology, such as personalized able on www.phrma.org.
medicine, are creating new opportunities to improve and
expand research into rare diseases and the development of
new treatments. While personalized medicine is just begin-
ning to impact patients, the Personalized Medicine Coalition
estimates that available personalized medicines, treatments
RARE DISEASES BIG IMPACT
and diagnostic products increased from 13 in 2006 to 72 by
2011. In just 10 years since the human genome was mapped,
452
real progress has been made.
The sequencing of the human genome and the analysis of

critical proteins in the blood have profoundly impacted bio-
pharmaceutical research and are yielding important new tools medicines
for understanding and treating a wide range of conditions. & vaccines in
These tools are proving critical for taking on rare diseases, development
which are often more complex than more common diseases.
Many rare diseases will require new tactics to find effective
treatments.
Researchers are increasingly able to identify much more tar-

geted patient populations and this new knowledge is allow-
ing clinicians to discover whether a patient is developing or Source: PhRMA, 2013 Rare Diseases Report
will develop an illness much earlier. Already researchers have

Key Issues
Prescription Drug User Fee Act PDUFA V Advances Regulatory

(PDUFA) Continues To be a Success Science
for Patients and Medical Innovation Through PDUFA V, FDA will have dedicated resources to
develop and apply new scientific tools and approaches to as-
Since 1992, the Prescription Drug User Fee Act (PDUFA)
sess the safety and effectiveness of new medicines.
has provided additional resources to FDA to help make
safe and effective new medicines available to patients in a • Additional FDA resources will help to better address
timely manner. In its first 20 years, PDUFA brought more submissions that involve pharmacogenomics and qualified
than 1,500 new medicines to Americans; increased FDA’s biomarkers.
staffing and resources; helped improve the consistency,
predictability and efficiency of FDA reviews; encouraged • FDA will support the development of treatments for rare
medical innovation and economic growth; and preserved diseases by increasing outreach to the patient commu-
and strengthened FDA’s high safety standards. PDUFA nity, providing specialized training for agency staff, and
has been reauthorized and amended four times since its developing guidance and policy related to advancing and
original passage in 1992—in 1997, 2002, 2007, and most facilitating the development of medicines for rare diseases.
recently in 2012 as PDUFA V.
• Dedicated FDA staff will evaluate and define best practic-
PDUFA V encompasses several provisions that will positively es for the conduct of meta-analyses and to inform guid-
impact research and development of treatments for rare ance on commonly accepted and standardized method-
diseases. ologies to be used in drug review and safety monitoring.
• Dedicated FDA staff will support the advancement of the

use of patient-reported outcomes and other outcomes
assessment tools.
Rare Disease Facts and Statistics

Here are a few statistics and facts to illustrate the breadth • In the United States, a condition is considered “rare” if
of the rare disease challenge in the United States and it affects fewer than 200,000 people.
worldwide.
• About 80 percent of rare diseases are genetic in origin,
• There are approximately 7,000 different types of rare and thus are present throughout a person’s life, even if
diseases and disorders, with more being discovered symptoms do not immediately appear.
each day.
• The prevalence distribution of rare diseases is
• 30 million people in the United States are living with skewed— 80 percent of all rare disease patients are
rare diseases. This equates to 1 in 10 Americans or affected by approximately 350 rare diseases.
10 percent of the population.
• According to the EveryLife Foundation for Rare Dis-
• It is estimated that 350 million people worldwide suf- eases, 95 percent of rare diseases lack a single FDA
fer from rare diseases. approved treatment.
• If all of the people with rare diseases lived in one ource: The Global Genes Project, a program of The
S
country, it would be the world’s third most populous R.A.R.E. Project
country.

Orphan Drugs in Development
Autoimmune Disorders
Product Name Sponsor Official FDA Designation* Development Status**
Actemra® Genentech treatment of systemic sclerosis to be Phase II

tocilizumab South San Francisco, CA a separate disease or condition from www.gene.com
localized scleroderma
apremilast Celgene treatment of Behcet’s disease Phase II

(CC-10004) Summit, NJ www.celgene.com
ARA290 Araim Pharmaceuticals treatment of neuropathic pain in Phase II

Ossining, NY patients with sarcoidosis www.araim.org
ARG201 arGentis Pharmaceuticals treatment of diffuse systemic sclerosis Phase II

(type 1 native bovine skin collagen) Memphis, TN www.argentisrx.com
caplacizumab Ablynx treatment of thrombotic Phase II

(ALX-0081) Ghent, Belgium thrombocytopenic purpura www.ablynx.com
Diamyd® Diamyd Medical treatment of type I diabetes with Phase III

autoimmune diabetes vaccine Stockholm, Sweden residual beta cell function www.diamyd.com
DiaPep277® Andromeda Biotech for use in type 1 diabetic mellitus Phase III
(peptide vaccine) Ness Ziona, Israel patients with residual beta-cell www.andromedabio.com
function
E5501 Eisai treatment of idiopathic Phase III

(avatrombopaq) Woodcliff Lake, NJ thrombocytopenic purpura www.eisai.com
Firdapse™ Catalyst Pharmaceutical treatment of Lambert-Eaton Phase III

amifampridine Coral Gables, FL myasthenic syndrome www.catalystpharma.com
(Breakthrough Therapy)
gevokizumab XOMA treatment of Behcet’s disease Phase III

(XOMA 052) Berkeley, CA (see also eye) www.xoma.com
Gilenya® Novartis Pharmaceuticals treatment of chronic inflammatory Phase III

fingolimod East Hanover, NJ demyelinating polyneuropathy www.novartis.com
IBC-VS01 Orban Biotech treatment of type 1 diabetes patients Phase I

(insulin B-chain vaccine) Brookline, MA with residual beta cell function www.orbanbiotech.com
Oralgam™ Latona Life Sciences treatment for juvenile rheumatoid Phase II completed
human gammaglobulin oral Scottsdale, AZ arthritis www.latonalifesciences.com
Pomalyst® Celgene treatment of systemic sclerosis Phase II

pomalidomide Summit, NJ (see also cancer, related) www.celgene.com
*The designation is issued by the FDA’s Office of Orphan Products Development while the drug is still in development. The designation makes the sponsor of the drug eligible
for entitlements under the Orphan Drug Act of 1983. The entitlements include seven years of marketing exclusivity following FDA approval of the drug for the designated use.
**For more information about a specific medicine or company in the report, please use the website provided.

Rare Diseases
Autoimmune Disorders
Product Name Sponsor Official FDA Designation Development Status
Prochymal® Osiris Therapeutics treatment of type 1 diabetes patients Phase II

remestemcel-L Columbia, MD with residual beta cell function www.osiris.com
(see also transplantation)
Simponi® Janssen Biotech treatment of sarcoidosis Phase II

golimumab Horsham, PA (see also digestive) www.janssenbiotech.com
Stelara® Janssen Biotech treatment of chronic sarcoidosis Phase II

ustekinumab Horsham, PA www.janssenbiotech.com
----------------------------------------- -----------------------------------------
treatment of primary biliary cirrhosis Phase II
www.janssenbiotech.com
teplizumab MacroGenics treatment of recent-onset type 1 Phase III

(anti-CD3 mAb) Rockville, MD diabetes www.macrogenics.com
Blood Disorders
fedratinib Sanofi US treatment of secondary and primary Phase III

Bridgewater, NJ myelofibrosis www.sanofi.com
----------------------------------------- -----------------------------------------
treatment of polycythemia vera Phase II
www.sanofi.com
human prothrombin complex Octapharma USA reversal of anticoagulation therapy in application submitted
concentrate Hoboken, NJ patients needing treatment of serious www.octapharma.us
or life-threatening bleeding and/or
needing urgent surgery or invasive
procedures
Jakafi® Incyte treatment of polycythemia vera Phase III

ruxolitinib Wilmington, DE (Fast Track) www.incyte.com
(see also cancer)
----------------------------------------- -----------------------------------------
treatment of essential Phase II
thrombocythemia www.incyte.com
LY2784544 Eli Lilly treatment of myeloproliferative Phase II

(gandotinib) Indianapolis, IN disorders (polycythemia vera, www.lilly.com
essential thrombocythemia and
myelofibrosis)

Blood Disorders
pacritinib Cell Therapeutics treatment of myeloproliferative Phase III

Seattle, WA disorders with the JAK2 V617F mutation www.celltherapeutics.com
simtuzumab Gilead Sciences treatment of myelofibrosis Phase II

(anti-LOXL2 mAb) Foster City, CA (see also cancer, respiratory) www.gilead.com
SPD602 Shire treatment of chronic iron overload in Phase II

(iron chelator) Wayne, PA patients with transfusion-dependent www.shire.com
anemias
Cancer
Abraxane® Celgene treatment of pancreatic cancer Phase III

paclitaxel protein-bound particles Summit, NJ (see also cancer, skin) www.celgene.com
for injection suspension,
(albumin-bound)
ABT-414 AbbVie treatment of glioblastoma multiforme Phase I

(EGFR ADC) North Chicago, IL (GBM) www.abbvie.com
ADI-PEG20 Polaris Pharmaceuticals treatment of hepatocellular Phase III

(pegylated arginine deiminase) San Diego, CA melanoma www.polarispharma.com
(see also cancer, skin)
ADXS-HPV Advaxis treatment of HPV-associated anal Phase I/II

Princeton, NJ cancer www.advaxis.com
AEZS-108 AEterna Zentaris treatment of ovarian cancer Phase II completed

(zoptarelin doxorubicin) South San Francisco, CA www.aezsinc.com
Afinitor® Novartis Pharmaceuticals treatment of hepatocellular Phase III

everolimus East Hanover, NJ carcinoma www.novartis.com
aldoxorubicin CytRx treatment of soft tissue sarcoma Phase II

Los Angeles, CA www.cytrx.com
-------------------------------------- -----------------------------------------
treatment of adenocarcinoma of the Phase II
pancreas www.cytrx.com
algenpantucel-L NewLink Genetics treatment of pancreatic cancer Phase III

Ames, IA (Fast Track) www.linkp.com

Cancer
alisertib Millennium Pharmaceuticals treatment of ovarian cancer Phase II completed

(MLN8237) Cambridge, MA (see also cancer, blood) www.millennium.com
AMG 102 Amgen treatment of gastric cancer including Phase III

(rilotumumab) Thousand Oaks, CA gastroesophageal junction www.amgen.com
adenocarcinoma
amrubicin Celgene treatment of small-cell-lung cancer Phase III

Summit, NJ www.celgene.com
Dainippon Sumitomo Pharma
Osaka, Japan
ARC-100 Archer Biosciences treatment of gliomas Phase II

(abeotaxane inhibitor) New York, NY www.archerbiosciences.com
----------------------------------------- -----------------------------------------
treatment of pediatric neuroblastoma Phase I/II
www.archerbiosciences.com
Archexin® Rexahn treatment of pancreatic cancer Phase II

antisense oligonucleotide Rockville, MD www.rexahn.com
astuprotimut-R GlaxoSmithKline treatment of MAGE-A3 positive Phase III

Rsch. Triangle Park, NC non-small-cell lung cancer www.gsk.com
ATR-101 Atterocor treatment of adrenocortical Phase I

Ann Arbor, MI carcinoma www.atterocor.com
AVO113 Activartis Biotech treatment of malignant glioma Phase II

(dendritic cell cancer Vienna, Austria www.activartis.com
immunotherapy)
Avastin® Genentech therapeutic treatment of patients application submitted

bevacizumab South San Francisco, CA with ovarian cancer www.gene.com
Azedra™ Progenics Pharmaceuticals treatment of neuroendocrine tumors Phase II

iobenguane I-131 Tarrytown, NY (Fast Track) www.progenics.com
BAY 94-9343 Bayer HealthCare Pharmaceuticals treatment of mesothelioma Phase I

(anti-mesothelin-ADC) Wayne, NJ www.bayerpharma.com
BC-819 BioCancell Therapeutics treatment of pancreatic cancer Phase II

(gene therapy) Jerusalem, Israel (Fast Track) www.biocancell.com

Cancer
CBP501 CanBas for use in combination with cisplatin Phase II

(peptide mimetic) Shizouka, Japan and pemetrexed for the treatment of www.canbas.co.jp
patients with mesothelioma
Ch14.18 mAb United Therapeutics treatment of neuroblastoma Phase III

Silver Spring, MD www.unithers.com
CO-1686 Clovis Oncology treatment of non-small-cell lung Phase I/II

(EGFR receptor antagonist) Boulder, CO cancer and mutations in the www.clovisoncology.com
epidermal growth factor receptor
Cotara® Peregrine Pharmaceuticals treatment of glioblastoma multiforme Phase II

TNT-1B mAB Tustin, CA and anaplastic astrocytoma www.peregrineinc.com
CP-613 Cornerstone Pharmaceuticals treatment of pancreatic cancer Phase I/II

Cranbury, NJ www.cornerstonepharma.com
CPP-1X Cancer Prevention Pharmaceuticals treatment of neuroblastoma Phase II

(eflornithine) Tucson, AZ www.canprevent.com
crenolanib AROG Pharmaceuticals treatment of malignant glioma Phase I

(CP-868-596) Dallas, TX (see also cancer, blood) www.arogpharma.com
CRS-207/GVAX Pancreas Aduro Biotech treatment of pancreatic cancer Phase II

(listeria monocytogenes) Berkeley, CA www.adutobiotech.com
CVac™ Prima Biomed treatment of ovarian cancer Phase II/III

intradermal cancer vaccine Sydney, Australia www.primabiomed.com
DCVax®-Brain Northwest Biotherapeutics treatment of primary brain malignant Phase III

dendritic cell vaccine Bethesda, MD cancer www.nwbio.com
EGEN-001 Expression Genetics treatment of ovarian cancer Phase II

Huntsville, AL www.egeninc.com
ENMD-2076 EntreMed treatment of ovarian carcinoma Phase II

Rockville, MD www.entremed.com
ensituximab Precision Biologics treatment of pancreatic cancer Phase I/II

(NPC-1C) Dallas, TX www.precision-biologics.com
Estybon® Onconova Therapeutics treatment of pancreatic cancer Phase III

rigosertib Newton, PA (see also cancer, blood) www.onconova.com

Cancer
etirinotecan pegol Nektar Therapeutics treatment of ovarian cancer Phase II

(NKTR-102) San Francisco, CA www.nektar.com
FANG™ Vaccine Gradalis treatment of ovarian cancer Phase II

autologous tumor cell vaccine Carrollton, TX (see also cancer, skin) www.gradalisinc.com
farletuzumab Eisai treatment of ovarian cancer Phase III

(MORAb-003) Woodcliff Lake, NJ www.eisai.com
G-202 GenSpera treatment of of hepatocellular Phase II

San Antonio, TX carcinoma www.genspera.com
G-203-2c Genus Oncology treatment of pancreatic cancer Phase I

(synthesized peptide) Vernon Hills, IL www.genusoncology.com
G-series prophage vaccine Agenus treatment of glioma Phase II

(vitespan) Lexington, MA www.agenusbio.com
GL-0810 Gliknik treatment of HPV-16-expressing head Phase II

(HPV-16 cancer vaccine) Baltimore, MD and neck squamous cell carcinoma www.gliknik.com
GL-0817 Gliknik treatment of MAGE-A3-expressing Phase II

(MAGE-A3 cancer therapeutic Baltimore, MD head and neck squamous cell www.gliknik.com
Trojan peptide vaccine) carcinoma
GliAtak® Advantagene treatment of malignant brain tumors Phase II

gene therapy Auburndale, MA www.advantagene.com
glufosfamide Eleison Pharmaceuticals for treatment of pancreatic cancer Phase III

St. Petersburg, FL (Fast Track) www.eleison-pharma.com
Havalen® Eisai treatment of advanced soft tissue Phase III

eribulin Woodcliff Lake, NJ sarcoma www.eisai.com
ICT-107 ImmunoCellular Therapeutics treatment of glioblastoma or brain Phase II

(dendritic cancer vaccine) Calabasas, CA stem glioma www.imuc.com
IMA901 immatics biotechnologies treatment of renal cell carcinoma in Phase III

(peptide vaccine) Tuebingen, Germany HLA-A*2-positive patients www.immatics.com
IMGN901 ImmunoGen treatment of small-cell lung cancer Phase II

(maytansinoid DM-1-conjugated Waltham, MA (see also cancer, blood) www.immunogen.com
mAb)

Cancer
IRX-2 IRX Therapeutics neoadjuvant therapy in patients with Phase II completed

New York, NY squamous cell carcinoma of the head www.irxtherapeutics.com
and neck (Fast Track)
Jakafi® Incyte treatment of pancreatic cancer Phase II

ruxolitinib Wilmington, DE (see also blood) www.incyte.com
lenvatinib Eisai treatment of follicullar, medullary, Phase III

Woodcliff Lake, NJ anaplastic cancer and metastatic or www.eisai.com
locally advanced papillary thyroid
Lutathera® Advanced Accelerator Applications treatment of gastro-entero-pancreatic Phase III

DOTA-Tyr3 octreotide Saint Genis Pouilly, France neuroendocrine tumors www.adacap.com
masitinib AB Science treatment of malignant Phase III

(AB-1010) Short Hills, NJ gastrointestinal stromal tumors www.ab-science.com
----------------------------------------- -----------------------------------------
treatment of patients with pancreatic Phase III
cancer www.ab-science.com
mibefradil Tau Therapeutics treatment of glioblastoma multiforme Phase I

Charlottesville, VA www.tautherapeutics.com
mifamurtide Millennium Pharmaceuticals treatment of osteosarcoma Phase III

Cambridge, MA www.millennium.com
milciclib Nerviano Medical Sciences treatment of thymic epithelial tumors Phase II

Nerviano, Italy www.nervianoms.com
MK-1775 Merck treatment of ovarian cancer Phase II

(WEE1 tyrosine kinase inhibitor) Whitehouse Station, NJ www.merck.com
MM-111 Merrimack Pharmaceuticals treatment of HER2-expressing Phase II

(bispecific antibody mAb) Cambridge, MA advanced adenocarcinoma of the www.merrimackpharma.com
stomach and gastroesophageal
junction
----------------------------------------- -----------------------------------------
treatment of HER2-expressing Phase II
adenocarcinoma of the esophagus www.merrimackpharma.com
MM-398 Merrimack Pharmaceuticals treatment of pancreatic cancer Phase III

(nanoliposomal irinotecan) Cambridge, MA www.merrimackpharma.com
MORAb-004 Eisai treatment of soft tissue sarcoma Phase II

Woodcliff Lake, NJ www.eisai.com

Cancer
MORAb-009 Eisai treatment of mesothelioma Phase II

(amatuximab) Woodcliff Lake, NJ www.eisai.com
MORAb-066 Eisai treatment of pancreatic cancer Phase I

Woodcliff Lake, NJ www.eisai.com
Multikine® CEL-SCI neoadjuvant therapy in patients with Phase III

leukocyte interleukin Vienna, VA squamous cell carcinoma of the head www.cel-sci.com
and neck
Nexavar® Bayer HealthCare Pharmaceuticals treatment of medullary thyroid application submitted

sorafenib Wayne, NJ cancer, anaplastic thyroid cancer, and www.bayerpharma.com
recurrent or metastatic follicular or
papillary thyroid cancer
NGF-hTNF MolMed treatment of malignant pleural Phase III

(recombinant fusion protein) Milan, Italy mesothelioma www.molmed.com
niraparib TESARO treatment of ovarian cancer Phase III

(ADP-ribose PARP inhibitor) Waltham, MA www.tesarobio.com
onartuzumab Genentech treatment of gastric cancer including Phase III

South San Francisco, CA gastroesophageal cancer www.gene.com
Oncophage® Agenus treatment of renal cell carcinoma Phase III

prophage cancer vaccine Lexington, MA (Fast Track) www.agenusbio.com
Opaxio® Cell Therapeutics treatment of glioblastoma multiforme Phase II

paclitaxel poliglumex Seattle, WA www.celltherapeutics.com
oregovomab Quest PharmaTech treatment of epithelial ovarian cancer Phase II

(B43.14 mAb) Edmonton, Canada www.questpharmatech.com
OSE2101 OSE Pharma treatment of non-small-cell lung Phase III

Paris, France cancer in patients expressing HLA-A2 www.osepharma.com
perifosine AEterna Zentaris treatment of neuroblastoma Phase I

Basking Ridge, NJ www.aezsinc.com
Perjeta® Genentech treatment of gastric cancer Phase III

pertuzumab South San Francisco, CA www.gene.com
pexastimogene devacirepvec Jennerex, Inc. treatment of hepatocellular Phase II

(Pexa-Vac; JX-594) San Francisco, CA carcinoma www.jennerex.com

Cancer
pimasertib EMD Serono treatment of pancreatic cancer Phase II

(MEK inhibitor-1) Rockland, MA www.emdserono.com
polyclonal antibody stimulator Cancer Advances treatment of gastric cancer Phase III
(G17DT immunogen) Durham, NC www.canceradvancesinc.com
----------------------------------------- -----------------------------------------
treatment of adenocarcinoma of the Phase III
pancreas www.canceradvancesinc.com
Poly-ICLC Emory University treatment of primary brain tumors Phase II

Atlanta, GA www.oncovir.com
Oncovir
Washington, DC
University of California
San Diego, CA
PV-10 Provectus Pharmaceuticals treatment of hepatocellular Phase I

(rose bengal disodium) Knoxville, TN carcinoma www.provectus.com
ramucirumab Eli Lilly treatment of gastric cancer application submitted

Indianapolis, IN www.lilly.com
-------------------------------------- -----------------------------------------
treatment of hepatocellular Phase III
carcinoma www.lilly.com
rindopepimut Celldex Therapeutics treatment of EGFRvIII-expressing Phase II/III

Phillipsburg, NJ glioblastoma multiforme (Fast Track) www.celldextherapeutics.com
rucaparib Clovis Oncology treatment of ovarian cancer Phase II

(PARP inhibitor) Boulder, CO www.clovisoncology.com
salirasib Kadmon treatment of panreatic cancer Phase II

(KD032) Warrendale, PA www.kadmon.com
simtuzumab Gilead Sciences treatment of pancreatic cancer Phase II

(anti-LOXL2 mAb) Foster City, CA (see also blood, respiratory) www.gilead.com
Somatuline® Ipsen Biopharmaceuticals treatment of symptoms associated Phase III

lanreotide acetate Basking Ridge, NJ with carcinoid syndrome www.ipsen.com
-------------------------------------- -----------------------------------------
treatment of neuroendocrine tumors Phase III
www.ipsen.com
SP1049C Supratek Pharma treatment of gastric cancer Phase II

Montreal, Canada www.supratek.com
squalamine Ohr Pharmaceutical treatment of ovarian cancer refractory Phase II

New York, NY or resistant to standard chemotherapy www.ohrpharmaceutical.com

Cancer
tegafur/gimeracil/oteracil Taiho Pharma USA treatment of gastric cancer Phase III

Princeton, NJ www.taiho.co.jp
telatinib ACT Biotech treatment of gastric cancer Phase II

(VEGFR/PDGFR/KIT inhibitor) San Francisco, CA www.actbiotech.com
telotristat etiprate Lexicon Pharmaceuticals management of symptoms of Phase III

(LX1032) The Woodlands, TX carcinoid syndrome associated with www.lexgen.com
carcinoid tumor
TH-302 EMD Serono treatment of soft tissue sarcoma Phase III

(hypoxia-activated prodrug) Rockland, MA www.emdserono.com
Threshold Pharmaceuticals www.thresholdpharm.com
South San Francisco, CA
ThermoDox® Celsion treatment of hepatocellular Phase III

lyso-thermosensitive liposomal Lawrenceville, NJ carcinoma (Fast Track) www.celsion.com
doxorubicin
Toca 511 & Toca FC combination Tocagen treatment of glioblastoma multiforme Phase I/II
San Diego, CA www.tocagen.com
trans sodium crocetinate Diffusion Pharmaceuticals treatment of glioblastoma in Phase I/II

(TSC) Charlottesville, VA conjunction with radiotherapy www.diffusionpharma.com
trebananib Amgen treatment of ovarian cancer Phase III

(AMG 386) Thousand Oaks, CA www.amgen.com
TVA-Brain-1 TVAX Biomedical treatment of primary central nervous Phase II

Lenexa, KS system malignancies www.tvaxbiomedical.com
Tykerb® GlaxoSmithKline treatment of ErbB2-positive Phase III

lapatinib Rsch. Triangle Park, NC esophageal cancer www.gsk.com
----------------------------------------- -----------------------------------------
treatment of ErbB2-positive Phase III
gastric cancer www.gsk.com
VAL-083 Del Mar Pharmaceuticals treatment of malignant gliomas Phase I/II

Vancouver, Canada www.delmarpharma.com
VB-111 Vascular Biogenics treatment of malignant glioma Phase I/II

Or Yehuda, Israel www.vblrx.com

Cancer
VB4-845 Viventia Biotech treatment of Ep-CAM-positive Phase II

(recombinant fusion protein) Winnipeg, Canada squamous cell carcinoma of the head www.viventia.com
and neck (Fast Track)
veliparib AbbVie treatment of epithelial ovarian cancer Phase II completed

North Chicago, IL in combination with DNA-damaging www.abbvie.com
agents
Votrient® GlaxoSmithKline treatment of ovarian cancer Phase III

pazopanib Rsch. Triangle Park, NC www.gsk.com
VS-6063 Verastem treatment of mesothelioma Phase II

(defactinib) Cambridge, MA www.verastem.com
Y-90 clivatuzumab Immunomedics treatment of pancreatic cancer Phase II

Morris Plains, NJ (Fast Track) www.immunomedics.com
Yondelis® Janssen Research & Development treatment of patients with ovarian Phase III
trabectedin Raritan, NJ cancer www.janssenrnd.com
----------------------------------------- -----------------------------------------
treatment of soft tissue sarcoma Phase III
www.janssenrnd.com
Zybrestat™ OXiGENE treatment of ovarian cancer Phase II

fosbreyabulin South San Francisco, CA (Fast Track) www.oxigene.com
Cancer, Blood
ACE-536 Acceleron Pharma treatment of myelodysplastic Phase II

(recombinant fusion protein) Cambridge, MA syndromes www.acceleronpharma.com
Celgene (see also genetic) www.celgene.com
Summit, NJ
Adcetris® Seattle Genetics treatment of patients with Phase III

brentuximab vedotin Bothell, WA peripheral T-cell lymphoma, not www.seattlegenetics.com
otherwise specified (Fast Track)

Cancer, Blood
AFM-13 Affimed Therapeutics treatment of Hodgkin lymphoma Phase I

(anti-CD30/CD16A mAb) Heidelberg, Germany www.affimed.com
alisertib Millennium Pharmaceuticals treatment of peripheral T-cell Phase III

(MLN8237) Cambridge, MA lymphoma www.millennium.com
(see also cancer)
allogeneic mesenchymal Mesoblast treatment of insufficient hematopoietic Phase III

precursor cells New York, NY stem cell production in patients with www.mesoblast.com
(stem cell therapy) hematologic malignancies who have
failed treatment with conventional
chemotherapy
AT-101 Ascenta Therapeutics treatment of chronic lymphocytic Phase II

(R-(-)-gossypol) Malvern, PA leukemia www.ascenta.com
AT9183 Astex Therapeutics treatment of acute myeloid leukemia Phase I/II completed
Dublin, CA www.astx.com
belinostat Spectrum Pharmaceuticals treatment of peripheral T-cell Phase II

Henderson, NV lymphoma (Fast Track) www.sppirx.com
BI-505 BioInvent International treatment of multiple myeloma Phase I

(anti-cellular adhesion Lund, Sweden www.bioinvent.com
molecule-1 mAb)
BiovaxID® Biovest International treatment of follicular lymphoma Phase III

dasiprotimut-T Tampa, FL (Fast Track) www.biovest.com
----------------------------------------- -----------------------------------------
treatment of mantle cell lymphoma Phase II
www.biovest.com
BL-8040 BioLineRx treatment of acute myeloid leukemia Phase II

(CXCR4 chemokine receptor) Jerusalem, Israel www.biolinerx.com
blinatumomab Amgen treatment of acute lymphocytic Phase II

Thousand Oaks, CA leukemia www.amgen.com
BP 100-1-01 Bio-Path treatment of chronic myelogenous Phase I

(liposomal Grb-2) Houston, TX leukemia www.biopathholdings.com
BT-062 Biotest Pharmaceuticals treatment of multiple myeloma Phase I/II

(indatuximab ravtansine) Boca Raton, FL www.biotestpharma.com

Cancer, Blood
CNDO-109 Coronado Biosciences treatment of acute myeloid leukemia Phase I/II

(activated allogeneic natural killer Burlington, MA www.coronadobiosciences.com
cells)
CPX-351 Celator Pharmaceuticals treatment of acute myeloid leukemia Phase III

(cytarabine/daunorubicin liposome Ewing, NJ www.celatorpharma.com
injection)
crenolanib AROG Pharmaceuticals treatment of acute myelogenous Phase II

(CP-868-596) Dallas, TX leukemia www.arogpharma.com
(see also cancer)
Dacogen® Eisai treatment of acute myeloid leukemia Phase II

decitabine Woodcliff Lake, NJ www.eisai.com
daratumumab Janssen Research & Development treatment of multiple myeloma Phase I/II
Raritan, NJ (Fast Track) www.janssenrnd.com
darinaparsin Solasia treatment of peripheral T-cell Phase I

(ZIO-101) Tokyo, Japan lymphoma www.ziopharm.com
ZIOPHARM Oncology
Boston, MA
dinaciclib Merck treatment of chronic lymphocytic Phase III

(MK-7965) Whitehouse Station, NJ leukemia www.merck.com
elotuzumab AbbVie treatment of multiple myeloma Phase III

North Chicago, IL www.abbvie.com
Bristol-Myers Squibb www.bms.com
Princeton, NJ
epratuzumab Immunomedics treatment of non-Hodgkin Phase II

Morris Plains, NJ lymphoma www.immunomedics.com
EPZ-5676 Epizyme treatment of acute lymphoblastic Phase I

(DOT1L protein inhibitor) Cambridge, MA leukemia (ALL) www.epizyme.com
Estybon™ Onconova Therapeutics treatment of myelodysplastic Phase III

rigosertib Newton, PA syndromes www.onconova.com
(see also cancer)
forodesine BioCryst Pharmaceuticals treatment of T-cell non-Hodgkin Phase II

(PNP inhibitor) Durham, NC lymphoma www.biocryst.com
Mundipharma www.mundipharma.com
Cambridge, United Kingdom

Cancer, Blood
ibrutinib Janssen Biotech treatment of chronic lymphocytic application submitted

Horsham, PA leukemia (Fast Track) www.janssenbiotech.com
Pharmacyclics (Breakthrough Therapy) www.pharmacyclics.com
Sunnyvale, CA ----------------------------------------- -----------------------------------------
treatment of mantle cell lymphoma Phase III
(Breakthrough Therapy) www.janssenbiotech.com
----------------------------------------- www.pharmacyclics.com
treatment of multiple myeloma -----------------------------------------
Phase II
www.janssenbiotech.com
www.pharmacyclics.com
idelalisib Gilead Sciences treatment of chronic lymphocytic Phase III

(PI3K delta inhibitor) Foster City, CA leukemia www.gilead.com
IMGN901 ImmunoGen treatment of multiple myeloma Phase I

(maytansinoid DM1-conjugated Waltham, MA (see also cancer) www.immunogen.com
mAb)
inotuzumab ozogamicin Pfizer treatment of B-cell acute Phase III

(CD22-targeted cytotoxic agent) New York, NY lymphoblastic leukemia www.pfizer.com
interleukin-21 Bristol-Myers Squibb treatment of stage II (T4), III or IV Phase I

(rIL-21) Princeton, NJ malignant melanoma www.bms.com
IPI-145 Infinity Pharmaceuticals treatment of follicular lymphoma Phase II

(PI3K δ/γ inhibitor) Cambridge, MA www.inpi.com
ISF35 Memgen treatment of chronic lymphocytic Phase II

(gene encoding chimeric CD40 San Diego, CA leukemia www.memgen.com
ligand)
lestaurtinib Teva Pharmaceutical treatment of acute myeloid leukemia Phase II

(CEP-701) North Wales, PA www.tevapharm.com
midostaurin Novartis Pharmaceuticals treatment of acute myeloid leukemia Phase III

(PKC412) East Hanover, NJ (see also other) www.novartis.com
milatuzumab Immunomedics treatment of chronic lymphocytic Phase I/II

Morris Plains, NJ leukemia www.immunomedics.com
MLN4924 Millennium Pharmaceuticals treatment of acute myelogenous Phase I

(NAE inhibitor) Cambridge, MA leukemia www.millennium.com
----------------------------------------- -----------------------------------------
treatment of myelodysplastic Phase I
syndromes www.millennium.com
MLN9708 Millennium Pharmaceuticals treatment of multiple myeloma Phase III

(ixazomib citrate) Cambridge, MA (see also other) www.millennium.com

Cancer, Blood
mocetinostat Mirati Therapeutics treatment of Hodgkin lymphoma Phase II

(MGCD0103) San Diego, CA www.mirati.com
mogamulizumab Kyowa Hakko Kirin Pharma treatment of patients with cutaneous Phase III
Princeton, NJ T-cell lymphoma www.kyowa-kirin-pharma.com
----------------------------------------- -----------------------------------------
treatment of adult T-cell leukemia/ Phase II
lymphoma (ATLL) www.kyowa-kirin-pharma.com
----------------------------------------- -----------------------------------------
treatment of peripheral T-cell Phase II
lymphoma www.kyowa-kirin-pharma.com
moxetumomab pasudotox AstraZeneca treatment of hairy cell leukemia Phase III

Wilmington, DE www.astrazeneca.com
MedImmune
Gaithersburg, MD
NiCord® Gamida Cell for use as hematopoietic support Phase I/II

stem cell therapy Jerusalem, Israel in patients with myelodysplastic www.gamida-cell.com
syndromes
obinutuzumab Genentech treatment of chronic lymphocytic application submitted

South San Francisco, CA leukemia www.gene.com
----------------------------------------- -----------------------------------------
treatment of diffuse large B-cell Phase III
lymphoma www.gene.com
ocaratuzumab MENTRIK Biotech treatment of follicular lymphoma Phase II

Dallas, TX www.mentrik.com
otlertuzumab Emergent BioSolutions treatment of chronic lymphocytic Phase II

(anti-CD37 mAb) Rockville, MD leukemia www.emergentbiosolutions.com
(TRU-016)
OXi4503 OXiGene treatment of acute myelogenous Phase I

(combretastatin A 1 diphosphate) South San Francisco, CA leukemia www.oxigene.com
panobinostat Novartis Pharmaceuticals treatment of multiple myeloma Phase III

East Hanover, NJ www.novartis.com
plitidepsin PharmaMar USA treatment of multiple myeloma Phase III

(cyclic depsipeptide) New York, NY www.pharmamar.com

Cancer, Blood
quizartinib Ambit Biosciences treatment of acute myeloid leukemia Phase II

(FLT3 inhibitor) San Diego, CA www.ambitbio.com
Revlimid® Celgene treatment of chronic lymphocytic Phase III

lenalidomide Summit, NJ leukemia www.celgene.com
----------------------------------------- -----------------------------------------
treatment of diffuse large B-cell Phase III
lymphoma www.celgene.com
----------------------------------------- -----------------------------------------
treatment of follicular lymphoma Phase III
www.celgene.com
sapacitabine Cyclacel treatment of acute myelogenous Phase III

Berkeley Heights, NJ leukemia www.cyclacel.com
----------------------------------------- -----------------------------------------
treatment of myelodysplastic Phase II
syndromes www.cyclacel.com
SHP-141 Shape Pharmaceuticals treatment of cutaneous T-cell Phase I

(HDACi inhibitor) Cambridge, MA lymphoma www.shapepharma.com
siltuximab Janssen Research & Development treatment of Castleman’s disease application submitted
Raritan, NJ www.janssenrnd.com
----------------------------------------- -----------------------------------------
treatment of multiple myeloma Phase II
www.janssenrnd.com
SL-401 Stemline Therapeutics treatment of acute myeloid leukemia Phase I/II

(recombinant fusion protein) New York, NY www.stemline.com
SNS01-T Senesco Technologies treatment of diffuse large B-cell Phase I/II

(DNA plasmid vector) Bridgewater, NJ lymphoma www.senesco.com
----------------------------------------- -----------------------------------------
treatment of mantle cell lymphoma Phase I/II
www.senesco.com
----------------------------------------- -----------------------------------------
treatment of multiple myeloma Phase I/II
www.senesco.com
StemEx® Gamida Cell for use as hematopoietic support in Phase III

carlecortemcel-L Jerusalem, Israel patients with relapsed or refractory www.gamida-cell.com
hematologic malignancies who are
receiving high-dose therapy
(Fast Track)

Cancer, Blood
Synribo® Teva Pharmaceutical treatment of myelodysplastic Phase II

omacetaine mepesuccinate North Wales, PA syndromes www.tevapharm.com
tabalumab Eli Lilly treatment of multiple myeloma Phase II/III

Indianapolis, IN www.lilly.com
Telintra® Telik treatment of myelodysplastic Phase II

ezatiostat Palo Alto, CA syndromes www.telik.com
tosedostat Cell Therapeutics treatment of acute myeloid leukemia Phase II

Seattle, WA www.celltherapeutics.com
Chroma Therapeutics
Oxon, United Kingdom
ublituximab TG Therapeutics treatment of chronic lymphocytic Phase I/II

(TG-1101) New York, NY leukemia www.tgtherapeutics.com
----------------------------------------- -----------------------------------------
treatment of nodal marginal zone Phase /II
lymphoma www.tgtherapeutics.com
----------------------------------------- -----------------------------------------
treatment of extranodal marginal Phase I/II
zone lymphoma (mucosa-associated www.tgtherapeutics.com
lymphatic tissue, MALT)
veltuzumab Immunomedics treatment of chronic lymphocytic Phase I/II

Morris Plains, NJ leukemia www.immunomedics.com
Vidaza® Celgene treatment of acute myeloid leukemia Phase III

azacitidine Summit, NJ www.celgene.com
vosaroxin Sunesis Pharmaceuticals treatment of acute myeloid leukemia Phase III

(SNS-595) South San Francisco, CA (Fast Track) www.sunesis.com
Xalkori® Pfizer treatment of anaplastic large-cell Phase I

crizotinib New York, NY lymphoma www.pfizer.com

Cancer, Related Conditions

Captisol-Enabled® melphalan Spectrum Pharmaceuticals high dose conditioning treatment Phase II/III
Henderson, NV prior to hematopoietic progenitor www/sppirx.com
(stem) cell transplantation
E-0316 Elorac topical treatment of pruritus Phase II

(naloxone topical) Vernon Hills, IL associated with mycosis fungoides www.eloracpharma.com
(Fast Track)
FT-1050 Fate Therapeutics enhancement of stem cell Phase II

(stem cell stimulant) San Diego, CA engraftment through ex-vivo www.fatetherapeutics.com
treatment of human allogeneic
hematopoietic stem cells (treatment
of neutropenia, thrombocytopenia,
lymphopenia, and anemia)
GC-4419 Galera Therapeutics prevention of radiation- or Phase I completed

(superoxide dismutase mimetic) Malvern, PA chemotherapy-induced oral mucositis www.galeratx.com
in cancer patients
LG631 Lentigen for bone marrow protection in the Phase I

(stem cell gene therapy) Gaithersburg, MD treatment of glioblastoma multiforme www.lentigen.com
momelotinib Gilead Sciences treatment of myelofibrosis Phase II

(JAK inhibitor) Foster City, CA www.gilead.com
Pomalyst® Celgene treatment of persons with myelo- Phase III

pomalidomide Summit, NJ proliferative neoplasm-associated www.celgene.com
myelofibrosis and anemia who are red
blood cell tranfusion dependent
(see also blood)
sodium thiosulfate (STS) Adherex Technologies prevention of platinum-induced Phase III

Rsch. Triangle Park, NC ototoxicity in pediatric patients www.adherex.com
Xerecept® Celtic Pharma treatment of peritumoral brain edema Phase III

corticotropin-releasing factor Hamilton, Bermuda www.celticpharma.com
Xgeva® Amgen treatment of hypercalcemia of Phase II

denosumab Thousand Oaks, CA malignancy www.amgen.com

Cancer, Skin
Abraxane® Celgene treatment of stage IIB to IV melanoma Phase III

paclitaxel protein-bound particles Summit, NJ (see also cancer) www.celgene.com
for injection suspension
(albumin-bound)
Adcetris® Seattle Genetics treatment of mycosis fungoides Phase III

brentuximab vedotin Bothell, WA (see also cancer, blood) www.seattlegenetics.com
ADI-PEG20 Polaris Pharmaceuticals treatment of invasive malignant Phase II

(pegylated arginine deiminase) San Diego, CA melanoma www.polarispharma.com
(see also cancer)
astuprotimut-R GlaxoSmithKline treatment of MAGE-A3-positive stages Phase III

Rsch. Triangle Park, NC IIB to IV malignant melanoma www.gsk.com
(see also cancer)
Cavatak™ Viralytics treatment of stage II (T4), stage III, Phase II

coxsackievirus A21 Sydney, Australia and stage IV melanoma www.viralytics.com
FANG™ Vaccine Gradalis treatment of stage IIB to IV melanoma Phase II

autologous tumor cell vaccine Carrollton, TX (see also cancer) www.gradalisinc.com
Marqibo® Spectrum Pharmaceuticals treatment of metastatic uveal Phase II

vincristine liposomal Henderson, NV melanoma www.sppirx.com
melapuldencel-T California Stem Cell treatment of stage IIIB through IV Phase II

(autologous dendritic cell vaccine) Irvine, CA metastatic melanoma www.californiastemcell.com
MK-3475 Merck treatment of stage IIB through IV Phase II

(lambrolizumab) Whitehouse Station, NJ malignant melanoma www.merck.com
nivolumab Bristol-Myers Squibb treatment of stage IIB to IV Phase III

(anti-PD-1 mAb) Princeton, NJ melanoma (Fast Track) www.bms.com
POL-103A Polynoma treatment of stage IIB to stage IV Phase III

(polyvalent melanoma vaccine) San Diego, CA melanoma www.polynoma.com
PV-10 Provectus Pharmaceuticals treatment of metastatic melanoma Phase II

(rose bengal disodium) Knoxville, TN (see also cancer) www.pvct.com
talimogene laherparepvec Amgen treatment of stage IIB-stage IV Phase III

Thousand Oaks, CA melanoma www.amgen.com
trametinib and dabrafenib GlaxoSmithKline treatment of stage IIB through IV application submitted
Rsch. Triangle Park, NC melanoma www.gsk.com

Cancer, Skin
veliparib AbbVie treatment of malignant melanoma Phase II

North Chicago, IL stages IIB through IV www.abbvie.com
(see also cancer)
Cardiovascular Diseases
ixmyelocel-T Aastrom Biosciences treatment of dilated cardiomyopathy Phase II

Ann Arbor, MI www.aastrom.com
plasmin (human) Grifols Therapeutics treatment of acute peripheral arterial Phase II

Los Angeles, CA occlusion www.grifolsusa.com
SRM003 Shire prevention of arteriovenous fistula or Phase II

Wayne, PA arteriovenous graft failure in patients www.shire.com
with end-stage renal disease
receiving hemodialysis or preparing
for hemodialysis (Fast Track)
tafamidis meglumine Pfizer treatment of symptomatic Phase III

New York, NY transthyretin (TTR) amyloid www.pfizer.com
cardiomyopathy
(see also genetic)
Digestive Diseases
Aes-210 AesRx topical treatment of children and Phase II

(clotrimazole) Newton, MA adults with pouchitis www.aesrx.com
alicaforsen Atlantic Healthcare treatment of pouchitis (Fast Track) Phase III

Essex, United Kingdom www.atlantichc.com
budesonide Meritage Pharma treatment of patients with Phase II

oral suspension San Diego, CA eosinophilic esophagitis www.meritagepharma.com
EUR-100 Aptalis Pharma US treatment of pediatric and adult Phase I/II

(fluticasone propionate) Bridgewater, NJ eosinophilic esophagitis www.aptalispharma.com

Digestive Diseases
Humira® AbbVie treatment of pediatric Crohn’s disease Phase III

adalimumab North Chicago, IL www.abbvie.com
Lialda® Shire treatment of ulcerative colitis in Phase I

mesalamine controlled release Wayne, PA pediatric patients www.shire.com
metronidazole 10% topical SLA Pharma topical treatment of active perianal Phase II
ointment Liestal, Switzerland Crohn’s disease www.slapharma.com
naltrexone low-dose TNI BioTech treatment of Crohn’s disease in Phase II

Bethesda, MD pediatric patients www.tnibiotech.com
orBec® Soligenix treatment of gastrointestinal Phase I

beclomethasone oral Princeton, NJ symptoms with chronic graft versus www.soligenix.com
host disease in patients
undergoing allogeneic hematopoietic
cell transplantation
reslizumab Teva Pharmaceutical treatment of children with Phase III

North Wales, PA eosinophilic esophagitis www.tevapharm.com
SGX203 Soligenix treatment of pediatric patients with Phase I

Princeton, NJ ulcerative colitis www.soligenix.com
Simponi® Janssen Biotech treatment of pediatric ulcerative Phase I

golimumab Horsham, PA colitis www.janssenbiotech.com
(see also autoimmune)
Soliris® Alexion Pharmaceuticals treatment of Shiga toxin-producing Phase II

eculizumab Cheshire, CT Escherichia coli hemolytic uremic www.alxn.com
syndrome
(see also eye)
Uceris® Santarus treatment of ulcerative colitis in Phase II

budesonide San Diego, CA pediatric patients aged 0 through www.santarus.com
16 years

Eye Disorders
CODA-001 CoDa Therapeutics treatment of persistent corneal Phase II

(antisense oligonucleotide) San Diego, CA epithelial defects www.codatherapeutics.com
EryDex EryDel treatment of ataxia-telangiectasia Phase I

erythrocyte-encapsulated Urbino, Italy www.erydel.com
dexamethasone
gevokizumab XOMA treatment of non-infectious Phase III

(XOMA 052) Berkeley, CA intermediate, posterior or pan uveitis, www.xoma.com
or chronic non-infectious anterior
uveitis
LIPO-102 Lithera treatment of symptomatic Phase II

(salmeterol/fluticasone) San Diego, CA exophthalmos associated with thyroid www.lithera.com
related eye disease
Mitosol® Mobius Therapeutics prevention of recurrence of pterygium application submitted

mitomycin St. Louis, MO after its surgical excision www.mobiustherapeutics.com
NT-501 CNTF Neurotech USA treatment of retinitis pigmentosa Phase II/III

(ciliary neurotrophic growth factor) Cumberland, RI (Fast Track) www.neurotechusa.com
----------------------------------------- -----------------------------------------
treatment of macular telangiectasia Phase I
type 2 (MacTel) www.neurotechusa.com
plasminogen concentrate Kedrion treatment of ligneous conjunctivitis Phase II/III

(human) Barga, Italy www.kedrion.com
QLT091001 QLT treatment of retinitis pigmentosa Phase I

(synthetic retinoid) Menlo Park, CA (see also genetic) www.qltinc.com
QPI-1007 Quark Pharmaceuticals treatment of ischemic optic Phase I

(synthetic double-stranded siRNA) Fremont, CA neuropathy www.quarkpharma.com
retinal pigment epithelium Advanced Cell Technology treatment of Stargardt’s macular Phase I/II
cell therapy Santa Monica, CA dystrophy www.advancedcell.com
RV001 River Vision treatment of active (dynamic) phase Phase II

(teprotumumab) New York, NY Grave’s orbitopathy
sirolimus opthalmic Santen treatment of chronic/refractory Phase III

(DE-109) Emeryville, CA anterior noninfectious uveitis, www.santen.com
noninfectious intermediate uveitis,
noninfectious panuveitis and
non-infectious uveitis affecting the
posterior of the eye (NICUPS)

Eye Disorders
Soliris® Alexion Pharmaceuticals treatment of neuromyelitis optica Phase II

eculizumab Cheshire, CT (see also digestive) www.alxn.com
StarGen™ Sanofi US treatment of Stargardt disease Phase I/II

gene therapy Bridgewater, NJ www.sanofi.com
UshStat® Sanofi US treatment of retinitis pigmentosa Phase I/II

gene therapy Bridgewater, NJ associated with Usher syndrome www.sanofi.com
1B gene defect
Vibex™/KXL™ System Avedro treatment of corneal ectasia following application submitted

riboflavin ophthalmic solution Waltham, MA refractive surgery www.avedro.com
----------------------------------------- -----------------------------------------
treatment of keratoconus application submitted
www.avedro.com
Genetic Disorders
AAV1-FS344 Milo Biotechnology treatment of Duchenne and Becker Phase I/II

(gene therapy-delivered myostatin Cleveland, OH muscular dystrophy www.milobiotechnology.com
inhibitor)
ABH001 Shire treatment of epidermolysis bullosa Phase III

human fibroblast-derived Wayne, PA (Fast Track) www.shire.com
dermal substitute
Abilify® Otsuka Pharmaceutical treatment of Tourette’s syndrome Phase III

aripiprazole Rockville, MD www.otsuka.com
ACE-536 Acceleron Pharma treatment of B-thalassemia Phase II

(recombinant fusion protein) Cambridge, MA (see also cancer, blood) www.acceleronpharma.com
Celgene www.celgene.com
Summit, NJ
Aeroquin® Aptalis Pharmaceuticals treatment of pulmonary infections Phase III

levofloxacin Birmingham, AL due to Pseudomonas aeruginosa and www.aptalispharma.com
other bacteria in patients with cystic
fibrosis
Aes-103 AesRx treatment of sickle cell disease Phase II

Newton, MA www.aesrx.com

Genetic Disorders
afamelanotide Clinuvel Pharmaceuticals treatment of erythropoietic Phase III

New York, NY porphyrias www.clinuvel.com
ALN-TTR02/GENZ438027 Alnylam Pharmaceuticals treatment of familial amyloidotic Phase II

(siRNA oligonucleotide) Cambridge, MA polyneuropathy www.alnylam.com
Sanofi US (Genzyme) www.sanofi.com
Bridgewater, NJ
alpha1-proteinase inhibitor Grifols Therapeutics treatment of cystic fibrosis Phase II

(human) Los Angeles, CA www.grifols.com
Alprolix™ Biogen Idec for the control and prevention of application submitted
recombinant factor IX fusion Weston, MA hemorrhagic episodes in patients www.biogenidec.com
protein with hemophilia B (congenital factor
IX deficiency or Christmas disease)
(Fast Track)
ALXN1101 Alexion Pharmaceuticals treatment of molybdenum cofactor Phase I

(cPMP replacement therapy) Cheshire, CT deficiency type A (MoCD) www.alxn.com
AMG 145 Amgen treatment of homozygous familial Phase III

(evolocumab) Thousand Oaks, CA hypercholesterolemia www.amgen.com
AMT060 uniQure treatment of hemophilia B Phase I/II

(factor IX gene therapy) Amsterdam, Netherlands www.uniqure.com
arbaclofen Seaside Therapeutics treatment of the behavioral Phase III

(STX209) Cambridge, MA abnormalities associated with fragile www.seasidetherapeutics.com
X syndrome
Arikace® Insmed treatment of bronchopulmonary Phase III

liposomal amikacin for inhalation Monmouth Junction, NJ Pseudomonas aeruginosa infections in www.insmed.com
cystic fibrosis patients
(see also infectious)
asfotase alfa Alexion Pharmaceuticals treatment of hypophosphatasia Phase II/III

Cheshire, CT (Fast Track) www.alxn.com

Genetic Disorders
ataluren PTC Therapeutics treatment of muscular dystrophy Phase III

(PTC124) South Plainfield, NJ resulting from premature stop www.ptcbio.com
mutations in the dystrophin gene
(Fast Track)
----------------------------------------- -----------------------------------------
for use in the treatment of cystic Phase III
fibrosis resulting from a nonsense www.ptcbio.com
(premature stopcodon) mutation in
the cystic fibrosis transmembrane
conductance regulatory gene
(Fast Track)
BAX111 Baxter International treatment of von Willebrand disease Phase III

(rhVWF) Deerfield, IL www.baxter.com
beloranib Zafgen treatment of Prader-Willi syndrome Phase II

Cambridge, MA www.zafgen.com
BMN-701 BioMarin Pharmaceutical treatment of Pompe disease Phase II

(IGF2-GAA) San Rafael, CA www.bmrn.com
Bronchitol® Pharmaxis for use to facilitate clearance of mucus application submitted

mannitol inhalation Exton, PA in patients with bronchiectasis and in www.pharmaxis.com
patients with cystic fibrosis at risk for
bronchiectasis (Fast Track)
CEQ508 Marina Biotech treatment of familial adenomatous Phase I/II

(RNA interference) Bothell, WA polyposis www.marinabio.com
ciprofloxacin dry powder Bayer HealthCare Pharmaceuticals management of pulmonary infection Phase II
inhalation (DPI) Wayne, NJ due to Pseudomonas aeruginosa in www.bayerpharma.com
Novartis Pharmaceuticals cystic fibrosis patients www.novartis.com
East Hanover, NJ
CPP-IX/sul Cancer Prevention Pharmaceuticals treatment of familial adenomatous Phase II

(eflornithine plus sulindac) Tucson, AZ polyposis www.canprevent.com
CSL654 CSL Behring treatment of patients with congenital Phase II/III

(rIX-FP) King of Prussia, PA factor IX deficiency (hemophilia B) www.cslbehring.com
CSL689 CSL Behring treatment and prophylaxis of Phase I

(rVIIa-FP) King of Prussia, PA bleeding episodes in patients with www.cslbehring.com
congenital hemophilia and inhibitors
to coagulation factor VIII or IX
----------------------------------------- -----------------------------------------
treatment of congenital factor VII Phase I
deficiency which includes treatment www.cslbehring.com
and prophylaxis of bleeding episodes
in patients with congenital factor VII
deficiency

Genetic Disorders
drisapersen GlaxoSmithKline treatment of Duchenne muscular Phase III

(GSK2402968) Rsch. Triangle Park, NC dystrophy www.gsk.com
Prosensa Therapeutics (Breakthrough Therapy)
Leiden, Switzerland
duvoglustat Amicus Therapeutics treatment of Pompe disease Phase II

(AT2220) Cranbury, NJ www.amicusrx.com
ecopipam Psyadon Pharmaceuticals symptomatic treatment of self Phase III

(PSYRX101) Germantown, MD injurious behaviors in patients with www.psyadonrx.com
Lesch-Nyhan disease
----------------------------------------- -----------------------------------------
treatment of Tourette’s syndrome in Phase II
children 0-16 years old www.psyadonrx.com
EDI1200 Edimer Pharmaceuticals treatment of X-linked hypohidrotic Phase II

Cambridge, MA ectodermal dysplasia (Fast Track) www.edimerpharma.com
eliglustat tartrate Sanofi US (Genzyme) treatment of Gaucher disease Phase III

(glucosylceramide synthase Bridgewater, NJ www.sanofi.com
inhibitor)
Eloctate™ Biogen Idec treatment of hemophilia A application submitted

recombinant human factor VIII-FC Weston, MA (Fast Track) www.biogenidec.com
EPI-743 Dainippon Sumitomo Pharma treatment of inherited mitochondrial Phase II/III

(vatiquinone) Osaka, Japan respiratory chain diseases www.ds-pharma.com
Edison Pharmaceuticals www.edisonpharma.com
Mountain View, CA
eteplirsen Sarepta Therapeutics treatment of Duchenne muscular Phase II

(antisense oligonucleotide) Cambridge, MA dystrophy (Fast Track) www.sareptatherapeutics.com
EXR-101 ExSAR treatment of GM-2 gangliosidoses Phase II

(pyrimethamine) Monmouth Junction, NJ (Tay-Sachs disease and Sandhoff www.exsar.com
disease)
EXR-202 ExSAR treatment of Gaucher disease Phase I

(ambroxol) Monmouth Junction, NJ www.exsar.com
GNE lipoplex Gradalis treatment of hereditary inclusion Phase I

Carrollton, TX body myopathy-2 www.gradalisinc.com
GSK2696273 GlaxoSmithKline treatment of severe combined Phase III

(ex-vivo stem cell gene therapy) Rsch. Triangle Park, NC immunodeficiency due to adenosine www.gsk.com
MolMed deaminase deficiency
Milan, Italy

Genetic Disorders
GZ402665 Sanofi US (Genzyme) treatment of acid sphingomyelinase Phase I

(rhASM) Bridgewater, NJ deficiency (Niemann-Pick disease) www.sanofi.com
halofuginone hydrobromide Halo Therapeutics treatment of Duchenne muscular Phase I/II

Newton, MA dystrophy www.halotherapeutics.com
HGT1110 Shire treatment of metachromatic Phase I/II

(cerebroside sulfatase) Wayne, PA leukodystrophy www.shire.com
HGT1410 Shire for treatment of Sanfilippo syndrome Phase I/II

(sulfamidase enzyme Wayne, PA (MPS IIIA) www.shire.com
replacement therapy)
HQK-1001 HemaQuest Pharmaceuticals treatment of beta thalassemia Phase II

San Diego, CA www.hemaquest.com
----------------------------------------- -----------------------------------------
treatment of sickle cell disease Phase II
www.hemaquest.com
human-cl rhFVIII Octapharma USA immune tolerance induction in Phase III

Hoboken, NJ hemophilia A patients with inhibitors www.octapharma.com
human coagulation factor X Bio Products Laboratory treatment of hereditary factor X Phase III
Herst, United Kingdom deficiency www.bpl.co.uk
idebenone Santhera Pharmaceuticals treatment of Duchenne muscular Phase III

Liestal, Switzerland dystrophy www.santhera.com
----------------------------------------- -----------------------------------------
treatment of mitochondrial Phase II
myopathy, encephalopathy, lactic www.santhera.com
acidosis with stroke-like episodes
syndrome (MELAS)
ISIS-TTRRX Isis Pharmaceuticals treatment of familial amyloid Phase III

(antisense oligonucleotides) Carlsbad, CA polyneuropathy (Fast Track) www.isispharm.com
L-glutamine Emmaus Medical treatment of sickle cell disease Phase III

Torrance, CA (Fast Track) www.emmausmedical.com
LCI699 Novartis Pharmaceuticals treatment of Cushing’s disease Phase II

(aldosterone synthase inhibitor) East Hanover, NJ www.novartis.com
LentiD® bluebird bio treatment of andrenoleukodystrophy Phase II/III

gene therapy Cambridge, MA www.bluebirdbio.com

Genetic Disorders
LentiGlobin® bluebird bio treatment of B-thalassemia major and Phase I/II

gene therapy Cambridge, MA intermediate www.bluebirdbio.com
LUM001 Lumena Pharmaceuticals treatment of alagille syndrome Phase II

(sodium bile acid cotransporter San Diego, CA www.lumenapharma.com
inhibitor) ----------------------------------------- -----------------------------------------
treatment of primary biliary cirrhosis Phase II
www.lumenapharma.com
----------------------------------------- -----------------------------------------
treatment of primary sclerosing Phase I
cholangitis www.lumenapharma.com
----------------------------------------- -----------------------------------------
treatment of progresssive familial Phase I
intrahepatic cholestasis www.lumenapharma.com
mavoglurant Novartis Pharmaceuticals treatment of fragile X syndrome Phase II/III

(AFQO56) East Hanover, NJ www.novartis.com
migalastat Amicus Therapeutics treatment of Fabry disease Phase III

Cranbury, NJ www.amicusrx.com
GlaxoSmithKline www.gsk.com
Rsch. Triangle Park, NC
Mirapex® Boehringer-Ingelheim Pharmaceuticals treatment of Tourette syndrome in Phase III completed

pramipexole Ridgefield, CT pediatric patients www.boehringer-ingelheim.com
MST-188 Mast Therapeutics treatment of sickle cell anemia Phase III

(purified poloxamer 188) San Diego, CA (Fast Track) www.masttherapeutics.com
NKTT120 NKT Therapeutics treatment of sickle cell disease Phase I

(rhIgG1k mAb) Waltham, MA www.nktrx.com
NN7999 Novo Nordisk routine prophylactic administration Phase III

(N9-GP) Princeton, NJ for prevention of bleeding in patients www.novonordisk.com
with hemophilia B (Christmas disease)
NovoThirteen® Novo Nordisk for the prevention of bleeding application submitted

catridecacog (rFXIII) Princeton, NJ associated with congenital FXIII www.novonordisk.com
deficiency
----------------------------------------- -----------------------------------------
treatment of congenital FXIII application submitted
deficiency www.novonordisk.com
OB-1 Baxter International treatment and prevention of episodic Phase III

(recombinant porcine factor VIII) Deerfield, IL bleeding in patients with inhibitor www.baxter.com
antibodies to human coagulation
factor VIII (Fast Track)

Genetic Disorders
PEG-PAL BioMarin Pharmaceutical treatment of hyperphenylalaninemia Phase III

(PEGylated recombinant San Rafael, CA www.bmrn.com
phenylalanine ammonia lyase)
pegylated carboxyhemoglobin Sangart treatment of acute painful sickling Phase I

San Diego, CA crises in patients with sickle cell www.sangart.com
disease
PF-05280602 Catalyst Biosciences routine prophylaxis to prevent Phase I

(rhFVIIa) South San Francisco, CA bleeding episodes in patients with www.catalystbiosciences.com
Pfizer hemophilia A and B patients with www.pfizer.com
New York, NY inhibitors
pradigastat Novartis Pharmaceuticals treatment of hypertriglyceridemia in Phase III

(LCQ908) East Hanover, NJ the setting of type I www.novartis.com
hyperlipoproteinemia, also known as
familial chylomicronemia syndrome
pridopidine Teva Pharmaceutical treatment of Huntington’s disease Phase II/III

North Wales, PA www.tevapharm.com
QLT091001 QLT treatment of Leber congenital Phase I

(synthetic retinoid) Menlo Park, CA amaurosis (LCA) due to inherited www.qltinc.com
mutations in RPE65 (encoding the
protein retinal pigment epithelial
protein 65) or LRAT (encoding the
enzyme lecithin:retinol
acyltransferase) genes
(see also eye)
rAAV1-CB-hAAT AGTC treatment of alpha1-antitrypsin Phase II

(adeno-associated Alachua, FL deficiency www.agtc.com
virus vector-mediated gene
therapy)
rAAV2-CB-hRPE65 AGTC treatment of type II Leber’s congenital Phase II

(gene therapy) Alachua, FL amaurosis www.agtc.com
RG2833 Repligen treatment of Friedreich’s ataxia Phase I

(HDAC3 inhibitor) Waltham, MA www.repligen.com
RG7090 Roche treatment of fragile X syndrome Phase II

Nutley, NJ www.roche.com

Genetic Disorders
RGN-137 topical RegeneRx Biopharmaceuticals treatment of epidermolysis bullosa Phase II completed

(thymosin beta 4) Rockville, MD www.regenerx.com
rivipansel GlycoMimetics treatment of vaso-occlusive crisis in Phase II

(GMI-1070) Gaithersburg, MD patients with sickle cell disease www.glycomimetics.com
Pfizer (Fast Track) www.pfizer.com
New York, NY
Ruconest® Santarus treatment of (acute attacks of) application submitted

conestat alfa San Diego, CA angioedema caused by hereditary www.santarus.com
or acquired C1-esterase inhibitor
deficiency
----------------------------------------- -----------------------------------------
prophylactic treatment of Phase II
angioedema caused by hereditary www.santarus.com
or acquired C1-esterase inhibitor
deficiency
SD-101 Scioderm treatment of skin blistering and Phase II

(allantoin) Durham, NC erosions associated with inherited www.sderm.com
epidermolysis bullosa
sebelipase alfa Synageva BioPharma treatment of lysosomal acid lipase Phase III
Lexington, MA deficiency (Fast Track) www.synageva.com
SelG2 Selexys Pharmaceuticals treatment of vaso-occlusive crisis in Phase II

(humanized IgG2 antibody) Oklahoma City, OK patients with sickle cell disease www.selexys.com
S-nitroglutathione N30 Pharmaceuticals management of cystic fibrosis Phase I

(N6022) Boulder, CO patients to improve airway clearance www.n30pharma.com
and to improve or stabilize pulmonary
function
Spiriva® HandiHaler® Boehringer Ingelheim Pharmaceuticals to improve pulmonary function in Phase III
tiotropium bromide Ridgefield, CT conjunction with standard therapy www.boehringer-ingelheim.com
in the management of patients with
cystic fibrosis
tafamidis meglumine Pfizer treatment of familial amyloid application submitted

New York, NY polyneuropathy www.pfizer.com
(see also cardiovascular)
TD-101 TransDerm treatment of pachyonychia congenita Phase I

(small interfering RNA) Santa Cruz, CA www.transderm.com

Genetic Disorders
UX001 Ultragenyx Pharmaceutical treatment of hereditary inclusion Phase II

(sailic acid extended release) Novato, CA body myopathy www.ultragenyx.com
Vimizim™ BioMarin Pharmaceutical treatment of mucopolysacharidosis application submitted

elosulfase alfa Novato, CA type IV A (Morquio A syndrome) www.bmrn.com
VX-809/ivacaftor Vertex Pharmaceuticals treatment of cystic fibrosis Phase III

Cambridge, MA (Fast Track) www.vrtx.com
Xenobilox® Sigma-Tau Pharmaceuticals treatment of cerebrotendinous application submitted

chenodeoxycholic acid Gaithersburg, MD xanthomatosis www.sigmatau.com
Zavesca® Actelion Pharmaceuticals treatment of the neurological application submitted

miglustat South San Francisco, CA manifestations of Niemann-Pick www.actelion.com
disease, type C
Growth Disorders
BMN-111 BioMarin Pharmaceutical treatment of achondroplasia Phase I

(modified recombinant human San Rafael, CA www.bmrn.com
C-type natriuretic peptide)
macimorelin acetate AEterna Zentaris diagnosis of growth hormone Phase III

(AEZS-130) South San Francisco, CA deficiency www.aezsinc.com
MOD-4023 PROLOR Biotech treatment of growth hormone Phase III

(hGH-CTP) Ness Ziona, Israel deficiency www.prolor-biotech.com
octreotide (oral) Chiasma for the oral treatment of acromegaly Phase I

Jerusalem, Israel www.chiasmapharma.com
Signifor® LAR Novartis Pharmaceuticals treatment of acromegaly Phase III

pasireotide East Hanover, NJ www.novartis.com
testosterone undecanoate (oral) SOV Therapeutics treatment of constitutional delay in Phase II

Morrisville, NC growth and puberty in adolescent www.sovtherapeutics.com
boys (14-17 yrs of age)

Growth Disorders
triptorelin pamoate Debiopharm treatment of central precocious Phase III

(Debio 8206 CPP) Lausanne, Switzerland puberty www.debiopharm.com
Infectious Diseases
AB-103 Atox Bio treatment of necrotizing soft tissue Phase II

(synthetic peptide) Ness Ziona, Israel infections (NSTI) www.atoxbio.com
Fast Track Drugs and Biologics (Fast Track)
North Potomac, MD
anthrax immune globulin (human) Cangene treatment of toxemia associated with Phase III
Winnipeg, Canada inhalational anthrax www.cangene.com
Anthrivig™ Emergent Biosolutions treatment of inhalation anthrax Phase III

anthrax immune globulin Rockville, MD disease (Fast Track) www.emergentbiosolutions.com
Arestvyr™ SIGA treatment of orthopoxvirus infections Phase II

tecovirimat New York, NY (Fast Track) www.siga.com
AriKace® Insmed treatment of bronchiectasis in Phase II completed

liposomal amikacin for inhalation Monmouth Junction, NJ patients with Pseudomonas www.insmed.com
aeruginosa or other susceptible
microbial pathogens
(see also genetic) -----------------------------------------
----------------------------------------- Phase II
treatment of infections caused by www.insmed.com
non-tuberculous mycobacteria
(Fast Track)
ASP0113 Astellas Pharma US prevention of clinically significant Phase III

(cytomegalovirus DNA vaccine) Northbrook, IL cytomegalovirus (CMV) viremia, www.astellas.com
(VCL-CB01) CMV disease and associated
complications in at-risk
hematopoietic cell transplant
populations
----------------------------------------- -----------------------------------------
prevention of clinically significant Phase II
cytomegalovirus (CMV) viremia, CMV www.astellas.com
disease and associated complications
in solid transplant populations

Infectious Diseases
AVI-7288 Sarepta Therapeutics prophylaxis following documented or Phase I

(antisense oligonucleotide) Cambridge, MA suspected exposure to Marburg virus www.sareptatherapeutics.com
(Fast Track)
Cayston® Gilead Sciences improvement of respiratory symptoms Phase III completed

aztreonam Foster City, CA in patients with bronchiectasis and www.gilead.com
gram-negative bacteria in the airways
Civacir® Biotest Pharmaceuticals prophylaxis of hepatitis C infection in Phase III

hepatitis C virus immune globulin Boca Raton, FL liver transplant recipients www.biotestpharma.com
(human)
delamanid Otsuka Pharmaceutical treatment of pulmonary tuberculosis Phase III

(OPC-67683) Rockville, MD www.otsuka.com
Dificid® Optimer Pharmaceuticals treatment of pediatric Clostridium Phase II

fidaxomicin Jersey City, NJ difficile infection www.optimerpharma.com
ETI-204 Elusys Therapeutics treatment of exposure to B. anthracis Phase I

(recombinant chimeric mAb) Pine Brook, NJ spores (Fast Track) www.elusys.com
isavuconazole Astellas Pharma US treatment of invasive aspergillosis Phase III

Northbrook, IL www.astellas.com
Basilea Pharmaceutica
Basel, Switzerland
maribavir ViroPharma treatment of clinically significant Phase II

Exton, PA cytomegalovirus viremia and disease www.viropharma.com
in at-risk patients (Fast Track)
MBX-400/cyclopropavir Microbiotix treatment of active cytomegalovirus Phase I

(nucleoside DNA polymerase Worcester, MA infections www.microbiotix.com
inhibitor)
miltefosine Paladin Therapeutics treatment of leishmaniasis application submitted

St-Laurant, Canada www.paladin-labs.com
MK-8228 Merck prevention of human Phase II

(letermovir) Whitehouse Station, NJ cytomegalovirus viremia and disease www.merck.com
in at-risk populations (Fast Track)
Nabi-HB® Biotest Pharmaceuticals prophylaxis against hepatitis B virus application submitted

hepatitis B immune globulin Boca Raton, FL reinfection liver transplant patients www.biotestpharma.com
(human)

Infectious Diseases
nifurtimox Bayer HealthCare Pharmaceuticals treatment of Chagas disease Phase I

Wayne, NJ (American typanosomiasis) caused www.bayerpharma.com
by T. cruzi
Nuartez™ Sigma-Tau Pharmaceuticals immediate treatment of malaria Phase III completed

artesunate injection Gaithersburg, MD www.sigmatau.com
US Army Medical Materiel
Development Activity
Ft. Detrick, MD
RiVax™ Soligenix prevention of ricin intoxication Phase I

ricin toxin pre-exposure vaccine Princeton, NJ www.soligenix.com
SQ109 Sequella treatment of tuberculosis Phase II

(ethylenediamine) Rockville, MD (Fast Track) www.sequella.com
sutezolid Sequella treatment of tuberculosis Phase II

Rockville, MD www.sequella.com
tafenoquine GlaxoSmithKline treatment of malaria Phase II

(8-aminoquinoline) Rsch. Triangle Park, NC www.gsk.com
TCN-202 Theraclone Sciences prevention of congenital Phase I

(mAb) Seattle, WA cytomegalovirus (CMV) infection www.theraclone-sciences.com
following primary CMV infection in
pregnant women
Thravixa™ Emergent BioSolutions for post-exposure prophylaxis and Phase I

human anthrax mAb Rockville, MD treatment of inhalation anthrax www.emergentbiosolutions.com
Valtorim® PharmAthene treatment of anthrax infection Phase I

anthrax anti-toxin mAb Annapolis, MD (Fast Track) www.pharmathene.com
vancomycin inhalation powder Savara Pharmaceuticals treatment of persistent methicillin- Phase II

Austin, TX resistant S. aureus lung infection in www.savarapharma.com
patients with cystic fibrosis
Neurological Disorders
ADX-N05 Aerial BioPharma treatment of narcolepsy Phase II

(neurotransmitter modulator) Morrisville, NC www.aerialbio.com
AmiKet™ Immune Pharmaceuticals treatment of postherpetic neuralgia Phase II completed

amitriptyline/ketamine Tarrytown, NY www.immunepharmaceuticals.com

arimoclomol ALS Association treatment of amyotrophic lateral Phase II/III

Richmond, VA sclerosis www.alsa.org
FDA Office of Orphan Products
Development
Silver Spring, MD
University of Miami
Miami, FL
ATI355 Novartis Pharmaceuticals treatment of acute spinal cord injury Phase I

(anti-Nogo-A mAb) East Hanover, NJ www.novartis.com
autologous bone marrow-derived TCA Cellular Therapy treatment of amyotrophic lateral Phase I
mesenchymal stem cells Covington, LA sclerosis www.tcacellulartherapy.com
BA-210 BioAxone BioSciences treatment of acute spinal cord injury Phase II

(recombinant fusion protein) Cambridge, MA (Fast Track) www.bioaxonebio.com
Captisol-enabled™ CURx Pharmaceuticals treatment of partial onset or primary Phase I

topiramate injection San Diego, CA generalized tonic-clonic seizures for www.ligand.com
Ligand Pharmaceuticals hospitalized epilepsy patients who are
La Jolla, CA unable to take oral topiramate
Circadin® Neurim Pharmaceuticals treatment of non-24-hour sleep-wake Phase III

melatonin controlled-release Tel-Aviv, Israel disorder in blind individuals without www.neurim.com
light perception
civamide Winston Laboratories treatment of postherpetic neuralgia Phase III

Vernon Hills, IL www.winstonlabs.com
----------------------------------------- -----------------------------------------
treatment of postherpetic neuralgia Phase II
of the trigeminal nerve www.winstonlabs.com
CPP-115 Catalyst Pharmaceutical Partners treatment of infantile spasms Phase I

(GABA-AT inhibitor) Coral Gables, FL www.catalystpharma.com
diazepam intranasal spray Acorda Therapeutics management of patients with acute Phase III
Ardsley, NY repetitive seizures www.acorda.com
Duopa® AbbVie treatment of late-stage Parkinson’s application submitted

levodopa/carbidopa intranuodenal North Chicago, IL disease (Fast Track) www.abbvie.com
Eladur™ DURECT relief of persistent pain associated Phase II

bupivacaine transdermal Cupertino, CA with postherpetic neuralgia www.durect.com
ganaxolone Marinus Pharmaceuticals treatment of infantile spasms Phase II

New Haven, CT www.marinuspharma.com

H.P. Acthar® Gel Quesctor Pharmaceuticals treatment of amyotrophic lateral Phase II

repository corticotropin injection Anaheim Hills, CA sclerosis www.questcor.com
subcutaneous
intravenous carbamazepine Lundbeck treatment of epilepsy patients who Phase III

Deerfield, IL cannot take anything by mouth (NPO) www.lundbeck.com
ISIS-SMNRX Biogen Idec treatment of spinal muscular atrophy Phase II

(antisense oligonucleotide) Weston, MA www.biogenidec.com
Isis Pharmaceuticals www.isispharm.com
Carlsbad, CA
midazolam intranasal Upsher-Smith Laboratories rescue treatment of seizures in Phase III

(USL-261) Maple Grove, MN patients who require control of www.upsher-smith.com
intermittent bouts of increased
seizure activity (e.g., acute repetitive
seizures, seizure clusters) (Fast Track)
NH001 NeuroHealing Pharmaceuticals for the treatment of patients in a Phase II

(apomorphine subcutaneous) Waban, MA vegetative state orminimally www.neurohealing.com
conscious state for up to 12 months
following a severe traumatic brain
injury (traumatic or spontaneous)
(Fast Track)
Northera® Chelsea Therapeutics treatment of neurogenic symptomatic application submitted

droxidopa Charlotte, NC orthostatic hypotension in patients www.chelseatherapeutics.com
with primary autonomic failure
(Parkinson’s disease, multiple system
atrophy, and pure autonomic failure),
dopamine-beta-hydroxylase
deficiency, and nondiabetic
autonomic neuropathy (Fast Track)
NP001 Neuraltus Pharmaceuticals for slowing the progression of Phase II

Palo Alto, CA amyotrophic lateral sclerosis www.neuraltus.com
NSI-566 Neuralstem treatment of amyotrophic lateral Phase II

(human spinal cord-derived neural Rockville, MD sclerosis www.neuralstem.com
stem cells)
NurOwn™ BrainStorm Cell Therapeutics treatment of amyotrophic lateral Phase II

(GDNF-producing stem cell New York, NY sclerosis www.brainstormcell.com
therapy)
ozanezumab GlaxoSmithKline treatment of amyotrophic lateral Phase II

(anti-nogo-A mAb) Rsch. Triangle Park, NC sclerosis www.gsk.com

PF-06687859 Pfizer treatment of spinal muscular atrophy Phase I

(mRNA decapping enzyme New York, NY (Fast Track) www.pfizer.com
inhibitor)
progesterone infusion BHR Pharma for early intervention in the treatment Phase III
(BHR-100) Herndon, VA of moderate to severe closed-head www.bhrpharma.com
traumatic brain injury (Fast Track)
RP103 Raptor Therapeutics treatment of Huntington’s disease Phase II/III

(cysteamine) Novato, CA www.raptorpharma.com
RTL-1000 Artielle ImmunoTherapeutics treatment of multiple sclerosis Phase I completed

(recombinant T-cell receptor San Mateo, CA patients who are both HLA-DR2- www.artielle.com
ligand) positive and autoreactive to myelin
oligodendrocyte glycoprotein residues
35-57
SEN196 Siena Biotech treatment of Huntington’s disease Phase I

(SIRT-1 inhibitor) Siena, Italy www.sienabiotech.com
tasimelteon Vanda Pharmaceuticals treatment of non-24 hour sleep/wake application submitted

Washington, DC disorder in blind individuals without www.vandapharma.com
light perception
tirasemtiv Cytokinetics treatment of amyotrophic lateral Phase II

South San Francisco, CA sclerosis (ALS) (Fast Track) www.cytokinetics.com
Vanquix® Pfizer management of selected, Phase III

diazepam auto-injector New York, NY refractory patients with epilepsy www.pfizer.com
on stable regimens of antiepileptic
drugs, who require intermittent
use of diazepam to control bouts of
increased seizure activity
Respiratory Disorders
Adempas™ Bayer HealthCare Pharmaceuticals treatment of pulmonary arterial application submitted

riociguat Wayne, NJ hypertension www.bayerpharma.com
AIR001 Aires Pharmaceuticals treatment of pulmonary arterial Phase II

(sodium nitrite) San Diego, CA hypertension www.airespharma.com

ARD-3150 Aradigm management of bronchiectasis Phase III

(liposomal ciprofloxacin) Hayward, CA www.aradigm.com
aviptadil THERAMetrics treatment of acute respiratory distress Phase I

(vasoactive intestinal peptide) Stans, Switzerland syndrome www.therametrics.com
beraprost 314d United Therapeutics treatment of pulmonary arterial Phase III

Silver Spring, MD hypertension www.unither.com
BMS-986202 Bristol-Myers Squibb treatment of idiopathic pulmonary Phase II

(LPA1 receptor antagonist) Princeton, NJ fibrosis www.bms.com
Esbriet® InterMune treatment of idiopathic pulmonary Phase III

pirfenidone Brisbane, CA fibrosis (Fast Track) www.intermune.com
FG-3019 FibroGen treatment of idiopathic pulmonary Phase II

(CTGF mAb inhibitor) San Francisco, CA fibrosis www.fibrogen.com
GeNosyl™ GeNO treatment of persistent pulmonary application submitted

nitric oxide Waltham, MA hypertension in newborns www.genollc.com
INOmax® Ikaria to reduce the risk of chronic lung Phase III

nitric oxide inhalation Hampton, NJ disease in premature neonates www.ikaria.com
----------------------------------------- -----------------------------------------
treatment of pulmonary arterial Phase I
hypertension www.ikaria.com
IW001 ImmuneWorks treatment of idiopathic pulmonary Phase I

(purified bovine type V collage) Indianapolis, IN fibrosis www.immuneworks.com
nintedanib Boehringer Ingelheim Pharmaceuticals treatment of patients with idiopathic Phase III
(triple kinase inhibitor) Ridgefield, CT pulmonary fibrosis www.boehringer-ingelheim.com
Opsumit® Actelion Pharmaceuticals treatment of pulmonary arterial application submitted

macitentan South San Francisco, CA hypertension www.actelion.com
PRM-151 Promedior treatment of idiopathic pulmonary Phase II

(recombinant human pentraxin-2 Lexington, MA fibrosis www.promedior.com
protein)
SAR156597 Sanofi US treatment of idiopathic pulmonary Phase II

(IL4/IL13 bi-specific antibody) Bridgewater, NJ fibrosis www.sanofi.com
selexipag Actelion Pharmaceuticals treatment of pulmonary arterial Phase III

South San Francisco, CA hypertension www.actelion.com

simtuzumab Gilead Sciences treatment of idiopathic pulmonary Phase II

(anti-LOXL2 mAb) Foster City, CA fibrosis www.gilead.com
(see also blood, cancer)
STX-100 Biogen Idec treatment of idiopathic pulmonary Phase II

(anti-integrin alphaVbeta6 mAb) Weston, MA fibrosis www.biogenidec.com
Surfaxin® Discovery Laboratories treatment of acute respiratory distress Phase II completed

lucinactant Warrington, PA syndrome in adults (Fast Track) www.discoverylabs.com
tralokinumab AstraZeneca treatment of idiopathic pulmonary Phase II

(anti-interleukin-13 mAb) Wilmington, DE fibrosis www.astrazeneca.com
MedImmune
Gaithersburg, MD
Skin
Debrase® MediWound debridement of acute, deep dermal Phase II completed

bromelain topical Yvane, Israel burns in hospitalized patients www.mediwound.com
StrataGraft™ Stratatech treatment of hospitalized patients Phase I/II

skin replacement therapy Madison, WI with complex skin defects resulting www.stratatechcorp.com
from partial and full thickness skin
burns requiring excision and grafting
Transplantation
anti-T-lymphocyte immune Fresenius Biotech prevention of graft versus host Phase III
globulin Waltham, MA disease (GVHD) www.fresenius-biotech.com
ASC-101 America Stem Cell to improve homing to bone Phase I/II

Floresville, TX (treatment of myeloablation) in www.americastemcell.com
patients receiving hematopoietic stem
cell transplantation

Transplantation
BB3 Angion Biomedica to improve renal function and prevent Phase II

(HGF mimetic) Uniondale, NY delayed graft function following renal www.angion.com
transplantation (Fast Track)
emricasan Conatus Pharmaceuticals treatment of patients undergoing Phase II

San Diego, CA solid organ transplantation (hepatic www.conatuspharma.com
fibrosis)
HSV-TK cell therapy MolMed immunotherapy for acceleration Phase III

Milan, Italy of T-cell reconstitution in patients www.molmed.com
undergoing allogeneic hematopoietic
stem cell transplantation
humanized IgG4 mAb Opsona Therapeutics prevention of ischemia/reperfusion Phase II

(OPN-305) Dublin, Ireland injury associated with solid organ www.opsona.com
transplantation
liposomal alpha REGiMMUNE prevention of graft versus host Phase I/II

galactosylceramide Santa Clara, CA disease www.regimmune.com
(RGI-2001)
Multistem® Athersys prophylaxis of graft versus host Phase I

stem cell therapy Cleveland, OH disease www.athersys.com
NX001 NephRx treatment of delayed graft function in Phase I

Kalamazoo, MI renal transplant recipients www.nephrx.com
----------------------------------------- -----------------------------------------
prevention of delayed graft function Phase I
in renal transplant recipients www.nephrx.com
Prochymal® Osiris Therapeutics treatment of acute graft versus host Phase III
remestemcel-L Columbia, MD disease (Fast Track) www.osiris.com
QPI-1002 Quark Pharmaceuticals prophylaxis of delayed graft function Phase II

(siRNA oligonucleotide) Fremont, CA in renal transplant patients www.quarkpharma.com
reparixin Dompe prevention of graft loss in pancreatic Phase II

(IL-8A/B receptor antagonist) Milan, Italy islet transplantation www.dompe.com

Transplantation
TOL101 Tolera Therapeutics prophylaxis of acute rejection of solid Phase I/II

(anti T-cell receptor murine mAb) Kalamazoo, MI organ transplantation www.tolera.com
TXA127 Tarix Pharmaceuticals to accelerate engraftment of Phase II

(angiotensin 1-7) Cambridge, MA hematopoietic cells (treatment of www.tarixpharma.com
neutropenia, thrombocytopenia,
lymphoma, and anemia) in
hematopoietic stem cell transplants
----------------------------------------- -----------------------------------------
treatment of patients requiring stem Phase I completed
cell transplantation to accelerate www.tarixpharma.com
the mobilization of hematopoietic
stem cells (CD34+) from the bone
marrow to the peripheral blood when
combined with a granulocyte
colony-stimulating factor
Other
Ampligen® Hemispherx Biopharma treatment of chronic fatigue Phase III completed

rintatolimid Philadelphia, PA syndrome www.hemispherxbiopharma.net
BIO300 Humanetics prevention of acute radiation Phase I

Minneapolis, MN syndrome www.humaneticscorp.com
BMN-190 BioMarin Pharmaceutical treatment of neuronal ceroid Phase I/II

(rhTPP1) San Rafael, CA lipofutscinosis type 2 www.bmrn.com
BYM338 Novartis Pharmaceuticals treatment of inclusion body myositis Phase II

(bimagrumab) East Hanover, NJ (Breakthrough Therapy) www.novartis.com
CYT-107 Cytheris treatment of progressive multifocal Phase II

(glycosylated recombinant Rockville, MD leukoencephalopathy www.cytheris.com
human interleukin-7)
Defitelo™ Gentium for the treatment of hepatic Phase III

defibrotide Villa Guardia, Italy veno-occlusive disease (Fast Track) www.sigmatau.com
Sigma-Tau Pharmaceuticals
Gaithersburg, MD

Other
DigiBind® Glenveigh Medical treatment of severe preeclampsia and Phase II

digoxin immune fab (ovine) Chattanooga, TN eclampsia (Fast Track) www.glenveigh.com
ELAD® Vital Therapies treatment of fulminant hepatic failure Phase III

immortalized human liver cells/ San Diego, CA (acute liver failure) www.vitaltherapies.com
extracorporeal liver assist device
entolimod Cleveland BioLabs prevention of death following a Phase I

Buffalo, NY potentially lethal dose of total body www.cbiolabs.com
irradiation during or after a radiation
disaster
fresolimumab Sanofi US treatment of primary focal segmental Phase II

(TGF-beta antagonist) Bridgewater, NJ glomerulosclerosis www.sanofi.com
human heterologous liver cells Cytonet treatment of urea cycle disorders Phase II
Durham, NC www.cytonetllc.com
Ilaris® Novartis Pharmaceuticals treatment of TNF receptor-associated Phase II

canakinumab East Hanover, NJ periodic syndrome (TRAPS) www.novartis.com
Kiacta™ Auven Therapeutics treatment of secondary amyloidosis Phase III

eprodisate New York, NY www.auventx.com
Lucassin® Ikaria treatment of hepatorenal syndrome application submitted

terlipressin Lebanon, NJ (Fast Track) www.ikaria.com
metreleptin Amylin Pharmaceuticals treatment of metabolic disorders application submitted

San Diego, CA secondary to lipodystrophy (Fast Track) www.bms.com
----------------------------------------- -----------------------------------------
treatment of leptin deficiency second- application submitted
ary to generalized lipodystrophy and www.bms.com
partial familial lipodystrophy
(Fast Track)
midostaurin Novartis Pharmaceuticals treatment of mastocytosis Phase II

(PKC412) East Hanover, NJ (see also cancer, blood) www.novartis.com
MLN9708 Millennium Pharmaceuticals treatment of systemic light chain (AL) Phase III
(ixazomib citrate) Cambridge, MA amyloidosis www.millennium.com
(see also cancer, blood)
Natpara® NPS Pharmaceuticals treatment of hypoparathyroidism Phase III

recombinant human parathyroid Bedminster, NJ www.npsp.com
hormone (rhPTH)

Other
Neo-Urinary Conduit™ Tengion treatment of bladder dysfunction Phase I

Winston-Salem, NC requiring incontinent urinary www.tengion.com
diversion
NT-100 Nora Therapeutics prevention of implantation failure Phase II

(rhGCSF) Palo Alto, CA www.noratherapeutics.com
obeticholic acid Intercept Pharmaceuticals treatment of primary biliary cirrhosis Phase III
(OCA) New York, NY www.interceptpharma.com
OCR-002 Ocera Therapeutics treatment of hyperammonemia and Phase II

(ornithine phenylacetate) San Diego, CA resultant hepatic encephalopathy www.ocerainc.com
(HE) in patients with acute liver
failure (Fast Track)
Octaplas LG® Octapharma USA treatment of thrombotic application submitted

human coagulation active plasma, Hoboken, NJ thrombocytopenic purpura www.octapharma.com
solvent/detergent treated
OrbeShield™ Soligenix prevention of death following a Phase I/II

beclomethasone dipropionate Princeton, NJ potentially lethal dose of total body www.soligenix.com
irradiation during or after a radiation
disaster (Fast Track)
Oxabact® OxThera treatment of primary hyperoxaluria Phase II/III

oxalobacter formigenes Stockholm, Sweden www.oxthera.com
PRT-201 Proteon Therapeutics prevention of arteriovenous fistula Phase II

(recombinant human type I Waltham, MA maturation and arteriovenous graft www.proteontherapeutics.com
pancreatic elastase) failure in patients with endstage
renal disease who are receiving
hemodialysis or preparing for
hemodialysis (Fast Track)
Ravicti™ Hyperion Therapeutics for intermittent or chronic treatment Phase II

glycerol South San Francisco, CA of patients with cirrhosis and any www.hyperiontx.com
grade hepatic encephalopathy
recilisib Onconova Therapeutics treatment of acute radiation Phase I

Newton, PA syndrome www.onconova.com
Samsca™ Otsuka Pharmaceuticals treatment of autosomal dominant application submitted

tolvaptan Rockville, MD polycystic kidney disease www.otsuka.com
(Fast Track)
SAR100842 Sanofi US treatment of patients with systemic Phase II

(LPA-1/LPA-3 antagonist) Bridgewater, NJ sclerosis www.sanofi.com

Other
Serelsa™ Autism Therapeutics treatment of autism Phase III

fluoxetine rapid dissolve New York, NY (Fast Track) www.autismtherapeutics.com
(AT001)
triheptanoin Ultragenyx treatment of fatty acid disorders Phase II

Novato, CA www.ultragenyx.com
uridine triacetate Wellstat Therapeutics an antidote in the treatment of Phase III

(PN401) Gaithersburg, MD 5-fluorouracil poisoning www.wellstattherapeutics.com
XEN402 Teva Pharmaceutical treatment of erythromelalgia Phase II

(sodium channel inhibitor) North Wales, PA www.tevapharm.com
Xenon Pharmaceuticals www.xenon-pharma.com
Burnaby, Canada
Xiaflex® Auxilium Pharmaceuticals treatment of Peyronie’s disease application submitted

collagenase clostridium Chesterbrook, PA www.auxilium.com
hystolyticum
The content of this report has been obtained through public, government (FDA’s Orphan Drug Product designation database) and
industry sources, and the Adis “R&D Insight” database based on the latest information. Report current as of September 23,
2013. The medicines in this report include medicines being developed by U.S. based companies conducting trials in the United
States and abroad, PhRMA-member companies conducting trials in the United States and abroad, and foreign companies con-
ducting clinical trials in the United States. The information in this report may not be comprehensive. For more specific informa-
tion about a particular product, contact the individual company directly or go to www.clinicaltrials.gov. The entire series of
Medicines in Development is available on PhRMA’s website.
A publication of PhRMA’s Communications & Public Affairs Department. (202) 835-3460
www.phrma.org | www.innovation.org | www.pparx.org
Provided as a Public Service by PhRMA. Founded in 1958 as the Pharmaceutical Manufacturers Association.
Copyright © 2013 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded if proper
credit is given.
Pharmaceutical Research and Manufacturers of America • 950 F Street, NW, Washington, DC 20004

Glossary
adenocarcinoma—Cancer of glandular muscles of the hips, pelvic area, thighs cutaneous—Pertaining to the skin.
tissue, or tumor of which gland-derived and shoulders. BMD is similar to Duch- cystic fibrosis—A genetic disorder of the
cells form gland-like structures. enne MD but often much less severe. exocrine glands (such as sweat glands or
adjuvant—A substance or drug that aids The disease progresses slowly and with kidneys) that causes abnormal mucous
another substance in its action. variability but can affect all voluntary secretions that obstruct glands and ducts
muscles. BMD primarily affects boys and in various organs.
alpha 1-proteinase inhibitor deficiency— men, who inherit the disease through
Although it is a rare condition, some their mothers. Most with BMD survive cytomegalovirus (CMV)—A DNA virus
people are congenitally deficient in alpha well into mid- to late adulthood. that can cause infection without symp-
1-proteinase inhibitor (or alpha 1-tryp- toms or with mild flu-like symptoms.
sin, a glycoprotein), which predisposes Breakthrough therapy—A designation
assigned by the U.S. Food and Drug diabetes—A chronic disease in which the
them to pulmonary emphysema early in body does not produce or properly use
life, even in the absence of exposure to Administration that is intended to
expedite the development and review insulin, a hormone that is needed to con-
substances (like cigarette smoke) that vert sugar, starches and other food into
interfere with lung-defense mechanisms. of drugs for serious or life-threatening
conditions. The criteria for breakthrough energy needed for daily life. Symptoms
amyotrophic lateral sclerosis (ALS)— therapy designation require preliminary may include excessive thirst, hunger,
Also known as Lou Gehrig’s disease, the clinical evidence that demonstrates the urination and weight loss. The cause
most common of the motor neuron dis- drug may have substantial improvement of diabetes continues to be a mystery,
eases, a group of rare disorders in which on at least one clinically significant end- although both genetics and environ-
the nerves that control muscular activity point over available therapy. A break- mental factors such as obesity and lack
degenerate within the brain and spinal through therapy designation conveys all of exercise appear to play roles. Type 1
cord causing weakness and wasting of of the Fast Track designation features, as diabetes, the more severe form, results
the muscles. well as more intensive FDA guidance on from the body’s failure to produce insu-
anaplastic thyroid carcinoma—An an efficient drug development program. lin, which “unlocks” the cells of the body,
aggressive, invasive form of cancer of allowing glucose to enter and fuel them.
carcinoma—Cancer. Squamous cell It is estimated that 5 percent to 10
the thyroid gland. It occurs most often carcinoma is one of the three most com-
in people over age 60. The cause is percent of Americans who are diagnosed
mon types of skin cancer, arising from with diabetes have type 1, which requires
unknown. Anaplastic cancer accounts the flattened, scale-like cells in the skin
for only about 1 percent of all thyroid insulin treatment.
and resulting primarily from long-term
cancers and is a very rare disease. exposure to the sun. Duchenne muscular dystrophy—An
application submitted—An application inherited disorder that involves rapidly
chronic fatigue syndrome—The symp- worsening muscle weakness. Other
for marketing has been submitted by the toms of this illness include debilitating
company to the Food and Drug Admin- muscular dystrophies get worse much
fatigue, interference with the ability to more slowly. Duchenne’s is caused by a
istration (FDA). concentrate, and, in some cases, a low- defective gene. Because of the way the
aspergillosis—Infection caused by asper- grade fever and swelling of the lymph disease is inherited, males are more likely
gillus, a fungus sometimes found in old nodes. Many possible causes have been to develop symptoms than are women.
buildings or decaying plant matter. implicated, but the true cause remains
unknown. epidermolysis bullosa—A rare, inherited
B-cell—A class of white blood cells im- condition in which blisters appear on
portant to the body’s immune system. Clostridium difficile—A bacterium that the skin after minor damage. It mainly
Becker muscular dystrophy (BMD)— produces an irritating toxin that causes a affects young children and has a wide
One of nine types of muscular dystro- form of colitis characterized by profuse, range of severity.
phy, a group of genetic, degenerative watery diar¬rhea with cramps and low-
Fabry disease—A genetic metabolic
diseases primarily affecting voluntary grade fever.
disorder that causes build-up of certain
muscles. It’s caused by an insufficient Crohn’s disease—A subacute chronic lipids. It becomes clinically apparent in
production of dystrophin, a protein that gastro¬intestinal disorder, involving the childhood and adolescence with fe-
helps keep muscle cells intact. Onset can small intestine, characterized by patchy ver, pain and small vascular tumors. It
occur during adolescence or adulthood. deep ulcers that may cause fistulas and a progresses to central nervous system
Symptoms include generalized weak- narrowing and thickening of the bowel. disturbances and renal and cardiac failure
ness and wasting, which first affects the Cushing disease— in mid-life.

Glossary
Fast Track—A process designed to Friedreich’s ataxia—An inherited graft versus host disease—In bone
facilitate the development and expedite disease that causes progressive dam- marrow transplantation, normal bone
the review of drugs to treat serious age to the nervous system resulting in marrow is used to replace malignant
diseases and fill an unmet medical need. symptoms ranging from gait disturbance or defective marrow. In an allogeneic
The status is assigned by the U.S. Food and speech problems to heart disease. transplantation, healthy marrow is taken
and Drug Administration. The purpose “Ataxia,” which refers to coordination from a donor; in an autologous trans-
is to get important new drugs to the problems such as clumsy or awkward plantation, the patient’s own healthy
patient earlier. Fast Track addresses a movements and unsteadiness, occurs in marrow is used. In graft vs. host disease,
broad range of serious diseases. Gener- many different diseases and conditions. a complication of such transplants, im-
ally, determining factors include whether The ataxia of Friedreich’s ataxia results mune system cells attack the transplant
the drug will have an impact on such from the degeneration of nerve tissue in recipient’s tissues.
factors as survival, day-to-day function- the spinal cord and of nerves that con- hematopoietic support—Helping the
ing, or the likelihood that the disease, if trol muscle movement in the arms and body to form blood or blood cells.
left untreated, will progress from a less legs. The spinal cord becomes thinner
severe condition to a more serious one. and nerve cells lose some of their myelin hemophilia A and B—Hemophilia A,
Filling an unmet medical need is defined sheath—the insular covering on all nerve the “clas¬sic” hemophilia, is a genetic
as providing a therapy where none exists cells that helps conduct nerve impulses. bleeding disorder due to deficiency of
or providing a therapy which may be The condition, although rare, is the the coagulation factor VIII. Hemophilia
potentially superior to existing therapy. most prevalent inherited ataxia, affecting B, or “Christmas” disease, is caused by
Once a drug receives Fast Track designa- about 1 in every 50,000 people in the deficiency of coagulation factor IX.
tion, early and frequent communication United States. hepatic—Related to the liver.
between the FDA and a drug company is FXIII deficiency (congenital)—A rare hepatitis—Inflammation of the liver
encouraged throughout the entire drug disease that affects 1 out of every 3-5 with accompanying liver cell damage
development and review process. The million people in the United States, or or death, caused most often by viral
frequency of communication assures that approximately 150 people. The condi- infection (e.g., types B and C), but also
questions and issues are resolved quickly, tion is characterized by blood that clots by certain drugs, chemicals or poisons.
often leading to earlier drug approval normally, but the clots are unstable, so Hepatitis may be either acute (of limited
and access by patients. bleeding recurs. FXIII deficiency can duration) or chronic (continuing).
fragile X syndrome—One of the most cause umbilical cord bleeding in some hepatocellular—Pertaining to the cells in
common causes of inherited mental newborns, soft tissue bruising, mucosal the liver.
retardation and neuropsychiatric disease bleeding and potentially fatal intracranial
in human beings, affecting as many as 1 hemorrhage (ICH). Studies have shown hepatocellular cancer/carcinoma—A
in 2,000 males and 1 in 4,000 females. that up to 60 percent of people with cancer that begins in the liver cells.
The syndrome is also known as FRAXA FXIII deficiency experience at least one hereditary angioedema—A rare but
(the fragile X chromosome itself) and as ICH during their lifetime. serious problem with the immune system
the Martin-Bell syndrome. However, the Gaucher disease—An inherited disease that is passed down through families. It
preferred name is fragile X syndrome. caused by a lack or deficiency of an en- is caused by low levels or improper func-
The characteristic features of the fragile zyme (glucocerebrosidase). Primarily af- tioning of a protein called C1 inhibitor,
X syndrome in boys include prominent or fects the liver, spleen and bone marrow. which affects the blood vessels. People
long ears, a long face, delayed speech, with hereditary angioedema can develop
large testes, hyperactivity, tactile defen- glioblastoma multiforme—The most rapid swelling of the hands, feet, limbs,
siveness, gross motor delays, and autis- common and most malignant of the as- face, intestinal tract, or airway (larynx or
tic-like behaviors. Much less is known trocytomas. The tumor grows so fast that trachea).
about girls with fragile X syndrome. it increases pressure in the brain, produc-
ing headaches, slowed thinking, and if HPV (human papillomavirus)—Viral
Only about half of all females who carry
severe enough, sleepiness and coma. agent of warts, believed to be conta-
the genetic mutation have symptoms
gious and usually harm-less, but it can
themselves. Of those, half are of normal glioma—A type of brain tumor arising lead to cervical cancer.
intelligence, and only one-fourth have from the supporting glial cells within
an IQ under seventy. Few fragile X girls the brain. Gliomas make up about 60 Huntington’s disease—Huntington’s
have autistic symptoms, although they percent of all primary brain tumors and chorea is an uncommon, inherited dis-
tend to be shy and quiet. are frequently malignant. ease in which degeneration of the basal

Glossary
ganglia (structures deep in the brain) tigue, and problems with the heart, eyes, the lymph nodes and spleen, multiply
results in chorea (rapid, jerky, involuntary and various other systems. unchecked. Lymphomas fall into two
movements) and dementia (progressive Juvenile rheumatoid arthritis—Refers to categories: One is called Hodgkin’s dis-
mental impairment). Symptoms do not arthritis or an arthritis-related condition ease, characterized by a particular kind
usually appear until the age of 35 to 50. (rheumatic disease) that occurs by age of abnormal cell. All others are called
hypercholesterolemia (homozygous 15 or younger. non-Hodgkin’s lymphomas, which vary in
familial)—An inherited metabolic dis- their malignancy according to the nature
Leber congenital amaurosis (LCA)—An and activity of the abnormal cells. B and
order resulting in an abnormal amount inherited retinal degenerative disease
of cholesterol in the blood. It can lead T-cell lymphomas are caused by prolifer-
characterized by severe loss of vision ation of the two principal types of white
to accelerated atherosclerosis and early at birth. A variety of other eye-related
heart attack. Dietary treatment seldom blood cells, called B- and T-lymphocytes.
abnormalities including roving eye move- Mycosis fungoides is a type of lym-
helps in these cases. ments, deep-set eyes, and sensitivity to phoma that primarily affects the skin of
hypophosphatasia—A rare, inherited bright light also occur with this disease. the buttocks, back or shoulders but can
disease that results in decreased activ- Some patients with LCA also experience also occur in other sites. The cause is
ity of the enzyme alkaline phosphatase, central nervous system abnormalities. unknown.
which assists in the metabolism of phos- Lesch-Nyhan syndrome (LNS)—A rare,
phate that is present in many tissues, in- melanoma—A cancer made up of pig-
inherited disorder caused by an enzyme mented skin cells.
cluding bones and teeth. The illness may (HPRT) deficiency. LNS is present at
occur during infancy or as an adult. The birth in baby boys. The lack of HPRT metastatic—Secondary cancers that
infantile form of hypophosphatasia is causes a build-up of uric acid in all body have spread from the primary or original
fatal in 50 percent of cases. Symptoms fluids, leading to symptoms such as cancer site.
of hypophosphatasia in infants include severe gout, poor muscle control, and mucositis—The swelling, irritation, and
poor feeding, failure to gain weight, moderate retardation, which appear in ulceration of the mucosal cells that line
failure to thrive, delayed development, the first year of life. A striking feature of the digestive tract. Mucositis can occur
loss of teeth, and bone pain. Adults LNS is self-mutilating behaviors–char- anywhere along the digestive tract from
who develop hypophosphatasia have acterized by lip and finger biting–that the mouth to the anus. It can be a very
a normal life expectancy. Symptoms in begin in the second year of life. Ab- troublesome and painful side effect of
adults include premature loss of teeth, normally high uric acid levels can cause chemotherapy.
fractures, and bone pain. sodium urate crystals to form in the multiple myeloma—A malignant condi-
idiopathic thrombocytopenia purpura— joints, kidneys, central nervous system, tion characterized by the uncontrolled
A condition in which there is destruc- and other tissues of the body, leading to proliferation and disordered function of
tion of blood platelets by the immune gout-like swelling in the joints and severe plasma cells (a type of white blood cell)
system. The reduced number of platelets kidney problems. Neurological symptoms in the bone marrow. It occurs in middle
may result in abnormal bleeding into the include facial grimacing, involuntary to old age and leaves patients vulnerable
skin (purpura) and other parts of the writhing, and repetitive movements of to increased infections and anemia.
body. the arms and legs similar to those seen
in Huntington’s disease. multiple sclerosis (MS)—Progressive
inherited mitochondrial diseases—A disease of the central nervous system in
group of systemic diseases caused by leukemia—A form of cancer involving which scattered patches of the covering
inherited or acquired damage to the mi- abnormally growing white blood cells, of nerve fibers (myelin) in the brain and
tochondria, which are small, energy-pro- which dominate the bone marrow and spinal cord are destroyed. Symptoms
ducing structures found in every cell in prevent it from making enough normal range from numbness and tingling to
the body that serve as the cells’ “power blood cells. This leaves the patient highly paralysis and incontinence.
plants.” When the mitochondria are not susceptible to serious infections, anemia
working properly, there is an energy and bleeding episodes. The cells increase neuroblastoma—A tumor of the adrenal
shortage within those areas of the body in the blood, interfering with the func- glands or sympathetic nervous system
that consume large amounts of energy tion of other organs. (the part of the nervous system respon-
such as the muscles, brain, and heart. sible for certain automatic body func-
lymphoma—Cancers in which the cells tions, such as the control of heart rate).
The result is often muscle weakness, fa- of lymphoid tissue, found mainly in Neuroblastomas are the most common

Glossary
extra-cranial (outside the skull) solid or completely eliminate this essential abnormal oxygen-carrying pigment
tumors of childhood. enzyme. Excessive amounts of glycogen called hemoglobin S, resulting in chronic,
neuropathic pain—Caused by disease, accumulated everywhere in the body, severe anemia and the characteristic
inflammation, or damage to the periph- but the cells of the heart and skeletal sickle shape of the red cell. Caused by
eral nerves, which connect the central muscles are the most seriously affected. mutation of the gene that codes for
nervous system (brain and spinal cord) to The symptoms of Pompe disease can hemoglobin.
the sense organs, muscles, glands, and vary widely in terms of age of onset and spinal cord injury—Damage to the spinal
internal organs. severity depending on the degree of cord which can cause loss of sensation,
enzyme deficiency. muscle weakness or paralysis.
neuropathy—Disease, inflammation, or
damage to the peripheral nerves, which postherpetic neuralgia—A burning pain systemic—Affecting the whole body.
connect the central nervous system to that may recur at the site of an attack of
shingles months or even years after the thalassemia—Not just one disease
the sense organs, muscles, glands, and but rather a complex series of genetic
internal organs. illness.
(inherited) disorders all of which involve
Parkinson’s disease—Chronic neurologic prophylaxis—Treatment intended to underproduction of hemoglobin, the
disease of unknown cause, characterized preserve health and prevent the spread indispensable molecule in red blood cells
by tremors, rigidity and an abnormal of disease. that carries oxygen.
gait. The most common variety is idio- pulmonary—Pertaining to the lungs. Tourette syndrome (TS)—A neurologi-
pathic Parkinson’s disease. pulmonary arterial hypertension—High cal disorder characterized by repetitive,
Phase 0—First-in-human trials conduct- blood pressure in the arteries supplying involuntary movements and vocalizations
ed in accordance with FDA’s 2006 guid- the lungs due to increased resistance to called tics. The early symptoms of TS are
ance on exploratory Investigational New blood flow through the lungs. typically noticed first in childhood, with
Drug (IND) studies designed to speed up renal—Relates to kidneys. the average onset between the ages of
development of promising drugs by es- 3 and 9. TS occurs in people from all
tablishing very early whether the tested respiratory distress syndrome (RDS)— ethnic groups; males are affected about
compound behaves in human subjects as Lung disorder of premature infants three to four times more often than
was anticipated from preclinical studies. characterized by respiratory distress and females. It is estimated that 200,000
cyanosis (lack of oxygen in blood). RDS Americans have the most severe form
Phase I—Safety testing and pharmaco- is caused by a deficiency of surfactant,
logical profiling in humans. of TS, and as many as 1 in 100 exhibit
a substance that coats the inner lining milder and less complex symptoms such
Phase II—Effectiveness and safety test- of the lungs and prevents them from col- as chronic motor or vocal tics. Although
ing in humans. lapsing during exhalation. TS can be a chronic condition with
Phase III—Extensive clinical trials to retinitis pigmentosa—Degeneration in symptoms lasting a lifetime, most people
demonstrate safety and efficacy in both eyes of the rods and cones of the with the condition experience their worst
humans. retina—the light-sensitive membrane that tic symptoms in their early teens, with
lines the inside of the back of the eye improvement occurring in the late teens
Pompe disease—A rare (estimated at
on which images are cast by the cornea and continuing into adulthood.
1 in every 40,000 births), inherited
and often fatal disorder that disables and lens. Usually has a genetic basis. ulcerative colitis—A chronic inflamma-
the heart and muscles. It is caused The first symptom is usually night blind- tion and ulceration of the lining of the
by mutations in a gene that makes an ness, progressing to a ring-shaped area colon and rectum. It causes bloody diar-
enzyme called alpha-glucosidase (GAA). of blindness that gradually extends to rhea and mainly involves the left colon.
Normally, the body uses GAA to break lessen the field of vision.
uveitis—Inflammation of the uvea, the
down glycogen, a stored form of sugar sickle cell anemia/disease—Inher- middle layer of the eye.
used for energy. But in Pompe disease, ited blood disorder in which red cells
mutations in the GAA gene reduce are abnormal in shape and contain an

The Drug Discovery, Development and Approval Process
Developing a new medicine takes an average of 10-15 years;

For every 5,000-10,000 compounds in the pipeline, only 1 is approved.
Drug Discovery and Development: A LON G , RISKY ROAD

DRUG DISCOVERY PRECLINICAL CLINICAL TRIALS FDA REVIEW LG-SCALE MFG
PHASE 4: POST-M AR K ET I N G SU RVEI L L A N C E

P R E - DI S COVERY
5,000 – 10,000 250 5

COMPOUNDS ONE FDA-
APPROVED
DRUG
PHASE PHASE PHASE

1 2 3
NDA SUBMIT T ED
IND SUBMIT T ED
NUMBER OF VOLUNTEERS
20 –80 100 – 300 1,000 – 3,000

0. 5 – 2
3 – 6 YEARS 6 – 7 Y EARS Y EARS
The Drug Development and Approval Process

The U.S. system of new drug approvals is in people. The IND shows results of previous ate statistically significant evidence to confirm
perhaps the most rigorous in the world. experiments; how, where and by whom the new its safety and effectiveness. They are the lon-
studies will be conducted; the chemical structure gest studies, and usually take place in multiple
It takes 10-15 years, on average, for an experi-
of the compound; how it is thought to work in sites around the world.
mental drug to travel from lab to U.S. patients,
the body; any toxic effects found in the animal
according to the Tufts Center for the Study of New Drug Application (NDA)/Biologic License
studies; and how the compound is manufac-
Drug Development. Only five in 5,000 com- Application (BLA). Following the completion
tured. All clinical trials must be reviewed and ap-
pounds that enter preclinical testing make it to of all three phases of clinical trials, a company
proved by the Institutional Review Board (IRB)
human testing. And only one of those five is analyzes all of the data and files an NDA or BLA
where the trials will be conducted. Progress
approved for sale. with FDA if the data successfully demonstrate
reports on clinical trials must be submitted at
both safety and effectiveness. The applications
On average, it costs a company $1.2 billion, least annually to FDA and the IRB.
contain all of the scientific information that the
including the cost of failures, to get one new
Clinical Trials, Phase I—Researchers test the company has gathered. Applications typically
medicine from the laboratory to U.S. patients,
drug in a small group of people, usually between run 100,000 pages or more.
according to a recent study by the Tufts Center
20 and 80 healthy adult volunteers, to evaluate
for the Study of Drug Development. Approval. Once FDA approves an NDA or BLA,
its initial safety and tolerability profile, deter-
the new medicine becomes available for physi-
Once a new compound has been identified in mine a safe dosage range, and identify potential
cians to prescribe. A company must continue
the laboratory, medicines are usually developed side effects.
to submit periodic reports to FDA, including
as follows:
Clinical Trials, Phase II—The drug is given any cases of adverse reactions and appropriate
Preclinical Testing. A pharmaceutical company to volunteer patients, usually between 100 and quality-control records. For some medicines,
conducts laboratory and animal studies to show 300, to see if it is effective, identify an optimal FDA requires additional trials (Phase IV) to
biological activity of the compound against the dose, and to further evaluate its short-term evaluate long-term effects.
targeted disease, and the compound is evalu- safety.
Discovering and developing safe and effective
ated for safety.
Clinical Trials, Phase III—The drug is given to a new medicines is a long, difficult, and expensive
Investigational New Drug Application (IND). larger, more diverse patient population, often process. PhRMA member companies invested an
After completing preclinical testing, a com- involving between 1,000 and 3,000 patients estimated $48.5 billion in research and develop-
pany files an IND with the U.S. Food and Drug (but sometime many more thousands), to gener- ment in 2012.
Administration (FDA) to begin to test the drug

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2013 REPORT

More Than 450 Medicines in Development for

This is, however, a time of great

for rare diseases. The medicines listed

in this report are either in clinical trials

or under review by the Food and Drug

Medicines in Development By Disease and Phase

Autoimmune Disorders 18 Application

2 Medicines in Development Rare Diseases 2013

ALS: Fighting a Devastating Disease—Amyotrophic lateral

Orphan Drug Act of 1983— RARE DISEASES BIG IMPACT

Source: National Institutes of Health

Medicines in Development Rare Diseases 2013 3

tumor-associated antigen mesothelin and allogeneic pancre-

Orphan Drug Approvals for

Seeking New Treatments for Children with a Rare Disease

4 Medicines in Development Rare Diseases 2013

cally, Ilaris was approved to treat two types of CAPS: familial

RARE DISEASES BIG IMPACT Patient Engagement

environment to help us meet the public health

RESEARCH PROGRESS HOPE

Medicines in Development Rare Diseases 2013 5

found genes associated with diseases such as myotonic dys-

FDA 400 APPROVED

Before a potential new treatment can be approved, it must

The sequencing of the human genome and the analysis of

Researchers are increasingly able to identify much more tar-

will develop an illness much earlier. Already researchers have

6 Medicines in Development Rare Diseases 2013

Prescription Drug User Fee Act PDUFA V Advances Regulatory

• Dedicated FDA staff will support the advancement of the

Rare Disease Facts and Statistics

Medicines in Development Rare Diseases 2013 7

Actemra® Genentech treatment of systemic sclerosis to be Phase II

apremilast Celgene treatment of Behcet’s disease Phase II

ARA290 Araim Pharmaceuticals treatment of neuropathic pain in Phase II

ARG201 arGentis Pharmaceuticals treatment of diffuse systemic sclerosis Phase II

caplacizumab Ablynx treatment of thrombotic Phase II

Diamyd® Diamyd Medical treatment of type I diabetes with Phase III

E5501 Eisai treatment of idiopathic Phase III

Firdapse™ Catalyst Pharmaceutical treatment of Lambert-Eaton Phase III

gevokizumab XOMA treatment of Behcet’s disease Phase III

Gilenya® Novartis Pharmaceuticals treatment of chronic inflammatory Phase III

IBC-VS01 Orban Biotech treatment of type 1 diabetes patients Phase I

Pomalyst® Celgene treatment of systemic sclerosis Phase II

8 Medicines in Development Rare Diseases 2013

Prochymal® Osiris Therapeutics treatment of type 1 diabetes patients Phase II

Simponi® Janssen Biotech treatment of sarcoidosis Phase II

Stelara® Janssen Biotech treatment of chronic sarcoidosis Phase II

teplizumab MacroGenics treatment of recent-onset type 1 Phase III

fedratinib Sanofi US treatment of secondary and primary Phase III

Jakafi® Incyte treatment of polycythemia vera Phase III

LY2784544 Eli Lilly treatment of myeloproliferative Phase II

Medicines in Development Rare Diseases 2013 9

pacritinib Cell Therapeutics treatment of myeloproliferative Phase III

simtuzumab Gilead Sciences treatment of myelofibrosis Phase II

SPD602 Shire treatment of chronic iron overload in Phase II

Abraxane® Celgene treatment of pancreatic cancer Phase III

ABT-414 AbbVie treatment of glioblastoma multiforme Phase I

ADI-PEG20 Polaris Pharmaceuticals treatment of hepatocellular Phase III

ADXS-HPV Advaxis treatment of HPV-associated anal Phase I/II

AEZS-108 AEterna Zentaris treatment of ovarian cancer Phase II completed

Afinitor® Novartis Pharmaceuticals treatment of hepatocellular Phase III

• Dedicated FDA staff will support the advancement of the