Once again thanks to the hard work of Mike O'Neill (@mikeoinsatx) I can offer BiotechDueDiligence readers an interesting resource. Below you'll find every company that received an Orphan Drug Designation from the FDA in 2012 along with the proposed clinical indication. Surely there is an investment gem in there somewhere?
(continue reading in expanded post)
(continue reading in expanded post)
2012 FDA designated "ORPHAN STATUS" LIST - Including sponsor and orphan designation:
Threshold Pharmaceuticals, Inc.
1 Treatment of soft tissue sarcoma
Albireo AB
1 Treatment of progressive familial intrahepatic cholestatis
2 Treatment of primary biliary cirrhosis
Onconova Therapeutics, Inc.
1 Prevention of acute radiation toxicity, also known as Acute Radiation Syndrome (ARS)
2 Treatment of acute radiation symdrome
Atterocor, Inc.
Treatment of adrenocortical carcinoma
Xenon Pharmaceuticals, Inc.
Treatment of erythromelalgia
Del Mar Pharmaceuticals (BC), Ltd
Treatment of malignant gliomas
ViroDefense, Inc.
Treatment of poliovirus infection.
LifeSplice Pharma LLC
Treatment of amyotrophic lateral sclerosis
Millennium Pharmaceuticals, Inc
Treatment of systemic light chain (AL) amyloidosis.
Hoffmann-La Roche, Inc.
Treatment of Fragile X Syndrome
Can-Fite BioPharma Ltd.
Treatment of hepatocellular carcinoma
Cortendo AB (HQ address)
Treatment of endogenous Cushing's syndrome
AbbVie, Inc.
Treatment of chronic lymphocytic leukemia
Sarepta Therapeutics, Inc.
1 Prophylaxis for patients following documented or suspected exposure to ebolavirus
2 Prophylaxis following documented or suspected exposure to marburg virus
Immatics Biotechnologies GmbH
Treatment of renal cell carcinoma in HLA-A*2 positive patients
ReGenX Biosciences LLC
Treatment of homozygous familial hypercholesterolemia
Lonza
Treatment of hospitalized patients with deep partial and full thickness burns requiring grafting.
Dutch Opthalmic Research Cente
To selectively stain the thickened internal limiting membrane, which has formed onto the inner side of the retina in vitreo-retinal disorders
Coronado Biosciences, Inc.
Treatment of acute myeloid leukemia
Senesco Technologies, Inc.
1 Treatment of diffuse large B cell lymphoma
2 Treatment of mantle cell lymphoma
Unicorn Pacific Corporation
Treatment of multiple myeloma
Immuno Tech, Inc.
Treatment of Kaposi sarcoma
HanAll BioPharma Co., Ltd.
Treatment of Behcet's disease
Surgeon General of the U. S. Army
Prevention of hemorrhagic fever with renal syndrome caused by Hantaan virus and Puumala virus.
AndromedA Biotech, LTD
For use in type 1 diabetic mellitus patients with residual beta-cell function
Intercell AG
Prevention of Japanese encephalitis virus in pediatric patients.
ERYTECH Pharma
Treatment of pancreatic cancer.
CTI Science, Inc.
Treatment of mercury toxicity
Biokine Therapeutics Ltd.
For use in combination with G-CSF to mobilize HSCs from the marrow to peripheral blood for collection for autologous or allogeneic transplantation
Arno Therapeutics, Inc.
1 Treatment of Schwannoma of the central nervous system
2 Treatment of meningioma
Edward A. Neuwelt, MD
Prevention of ototoxicity caused by platinum-based chemotherapeutic agents used to treat pediatric cancers
JSK Therapeutics, Inc.
Treatment of acute myeloid leukemia
EcoPharm Corporation
Treatment of idiopathic thrombocytopenic purpura
PharmaIN Corporation
Treatment of refractory ascites due to all etiologies except for cancer
Apogenix GmbH
Treatment of Myelodysplastic Syndrome
Pfizer, Inc.
Routine prophylaxis to prevent bleeding episodes in patients with hemophilia A and B patients with inhibitors
AbbVie, Inc.
Treatment of glioblastoma multiforme (GBM).
Salupont Consulting Ltd
Treatment of severe pre-eclampsia
Rottapharm S.p.A.
Prevention of recurrent hepatitis C in liver transplant patients
Luitpold Pharmaceuticals, Inc.
Treatment of uremic and non-uremic calciphylaxis
Quark Pharmaceuticals, Inc.
Treatment of ischemic optic neuropathy
OncoTherapy Science, Inc.
Treatment of soft tissue sarcoma
Sigma-Tau Pharmaceuticals, Inc.
Treatment of Fragile X syndrome
Genable Technologies Limited / Smurfit Institute of Genetics
treatment of retinitis pigmentosa
Laboratorios del Dr. Esteve, S.A.
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Milo Biotechnology
Treatment of Duchennes and Becker's muscular dystrophy
Athersys, Inc.
Treatment of MPS-1, including Hurler syndrome
Boehringer Ingelheim Pharmaceuticals, Inc.
Treatment of epidermal growth factor receptor mutation positive non-small cell lung cancer
Niche Therapeutics, LLC
Treatment of ST-elevation myocardial infarcted patients who are undergoing primary percutaneous coronary intervention
Niche Therapeutics, LLC
Treatment of ST-elevation myocardial infarcted patients who are undergoing primary percutaneous coronary intervention
Millennium Pharmaceuticals, Inc.
Treatment of peripheral T-cell lymphoma
Neurotech USA, Inc.
Treatment of macular telangiectasia type 2 (MacTel)
Grifols Therapeutics, Inc.
Treatment of cystic fibrosis
Morphotek, Inc.
Treatment of mesothelioma
Recepta Biopharma S.A.
Treament of ovarian cancer
Bayer HealthCare Pharmaceuticals, Inc.
Treatment of mesothelioma
Isis Pharmaceuticals, Inc.
Treatment of familial amyloid polyneuropathy
LipimetiX Development, LLC
Treatment of homozygous familial hypercholesterolemia
bluebird bio, Inc.
Treatment of andrenoleukodystrophy
Duke University
Treatment of pediatric (0-16 yrs old inclusive) cerebral palsy due to acquired brain injury
Soligenix, Inc
Prevention of death following a potentially lethal dose of total body irradiation during or after a radiation disaster
Guochuan Emil Tsai, MD, PhD
University of California, Los Angeles
Treatment of pediatric schizophrenia
Gradalis, Inc.
Treatment of stage IIB to IV melanoma
Seattle Genetics, Inc.
Treatment of mycosis fungoides
Luitpold Pharmaceuticals, Inc.
Treatment of seizure prolongation in patients undergoing electroconvulsive therapy
Novartis Pharmaceuticals Corporation
Treatment of TNF-receptor associated periodic syndrome (TRAPS)
Orenova Group, LLC
Treatment of perforating diseases, including Kyrle's disease, reactive perforating collangenosis, perforating folliculitis and elastosis perforans serpiginosa
Hillhurst Biopharmaceuticals, Inc.
Treatment of sickle cell disease
SK Life Science, Inc.
Management of patients with infantile spasms
GANYMED Pharmaceuticals AG
Treatment of gastric cancer
Baxter Healthcare Corporation
Prophylactic use to prevent or reduce the frequency of bleeding episodes in patients with hemophilia B (routine prophylaxis in patients where there is no evidence or suspicion of bleeding)
OXiGene, Inc.
Treatment of acute myelogenous leukemia
Stephen G. Kaler, M.D.
Head, Unit on Human Copper Metabolism
Treatment of Menkes disease
AROG Pharmaceuticals, LLC
Treatment of acute myelogenous leukemia
Pfizer
1 Treatment of neuroblastoma
2 Treatment of anaplastic large cell lymphoma
Lumos Pharma
Treatment of creatine transporter deficiency
Eagle Pharmaceuticals, Inc.
Treatment of heat stroke
Luitpold Pharmaceuticals, Inc.
Treatment of trigeminal neuralgia
EryDel S.p.A.
Treatment of ataxia-telangiectasia
S. Cotter
Treatment of Prader Willi Syndrome
Acorda Therapeutics, Inc.
Management of patients with acute repetitive seizures
Glenveigh Medical, LLC
Treatment of severe preeclampsia and eclampsia
Proteo Biotech AG
Treatment of pulmonary arterial hypertension
Thrombologic
Treatment of purpura fulminans
Eisai, Inc.
Treatment of advanced soft tissue sarcoma
Novartis Pharmaceuticals Corporation
Treatment of hepatocellular carcinoma
Invenux, LLC
Treatment of sickle cell disease
ArsNova Partners, LLC
For use in magnetic resonance imaging to assess, and monitor treatment of solid tumor malignancies previously diagnosed in pediatric patients (age 16 years and younger)
XOMA (US) LLC
Treatment of non-infectious intermediate, posterior or pan uveitis, or chronic non-infectious anterior uveitis
Biodel, Inc.
Prevention of hypoglycemia in the congenital hyperinsulinism population
Cytheris, Inc.
Treatment of progressive multifocal leukoencephalopathy
Janssen Biotech, Inc.
1 Treatment of sarcoidosis
2 Treatment of pediatric ulcerative colitis
Cadila Pharmaceuticals Limited
Treatment of non-small cell lung cancers that express desmocollin-3
Promethera Biosciences
Treatment of urea cycle disorders
Promethera Biosciences
Treatment of Crigler-Najjar syndrome
Laboratoris Sanifit, S.L.
Treatment of calciphylaxis
Onclave Theraeputics Limited
Treatment of AA amyloidosis and AL amyloidosis
Galileo Research srl
Treatment of ovarian cancer
OCTAPHARMA USA, Inc.
Immune tolerance induction in hemophilia A patients with inhibitors
FibroGen, Inc.
Treatment of idiopathic pulmonary fibrosis
jCyte, Inc.
Treatment of retinitis pigmentosa
Merck, Sharp & Dohme Corp.
Treatment of Stage IIB through IV malignant melanoma
Pharmacyclics, Inc.
1 Treatment of chronic lymphocytic leukemia
2 Treatment of mantle cell lymphoma
3 Treatment of chronic lymphocytic leukemia (CLL).
Algorithum Sciences, LLC
Treatment of pulmonary arterial hypertension
AbbVie, Inc.
Treatment of ovarian cancer
Medgenics, Inc.
Treatment of chronic hepatitis D
Eisai, Inc.
Treatment of follicullar, medullary, anaplastic, and metastatic or locally advanced papillary thyroid cancer
Astellas Pharma Global Development, Inc.
Treatment of adrenocortical carcinoma
Aradigm Corporation
For the management of cystic fibrosis
REGiMMUNE Corporation
Prevention of graft-versus-host disease
Pharma Mar USA Inc.
Treatment of ovarian cancer
PCUT BioPartners, Inc.
Treatment of amyotropic lateral sclerosis
Premacure AB
Prevention of retinopathy of prematurity in premature infants born at risk for the disease
Icon Bioscience, Inc.
Treatment of retinoblastoma
OncoTx, LLC
Treatment of Stage IIB through IV melanoma
Nu Science Trading, LLC
Treatment of calciphylaxis
Nova Laboratories Limited
Treatment of acute lymphoblastic leukemia in pediatric patients
Fera Pharmaceuticals, LLC
Treatment of methemoglobinemia
Luitpold Pharmaceuticals, Inc.
Treatment of congenital and acquired methemoglobinemia
Provepharm SAS
Treatment of hereditary methemoglobinemia
Laboratoire HRA Pharma
Treatment of Cushing's syndrome
Meridian Medical Technologies, Inc.
Treatment of nerve agent-induced seizures
Nerviano Medical Sciences S.r.l.
Treatment of thymic epithelial tumors.
Gencia Corporation
Treatment of inherited mitochondrial respiratory chain disease
APEIRON Biologics AG
Treatment of neuroblastoma.
Luitpold Pharmaceuticals, Inc.
Treatment of Myasthenia Gravis
GeNO, LLC
Treatment of persistent pulmonary hypertension in newborns
Genentech, Inc
1 Treatment of chronic lymphocytic leukemia
2 Treatment of diffuse large B cell lymphoma
Aerial BioPharma, LLC
Treatment of narcolepsy
Terra Biological LLC
Treatment of gliomas
Cell Therapeutics, Inc.
Treatment of glioblastoma multiforme
Novartis Pharmaceuticals Corporation
Treatment of multiple myeloma
PharmaEssentia Corporation
Treatment of polycythemia vera
Keck Graduate Institute of Applied Life Sciences
Treatment of Behcet's disease
Eisai, Inc.
Treatment of Lennox-Gastaut Syndrome
Heart Metabolics, Ltd.
Treatment of moderate to severe symptomatic (NYHA class III or IV) hypertrophic cardiomyopathy
Luitpold Pharmaceuticals, Inc.
Treatment of Tetralogy of Fallot
TRB Chemedica International S.A.
Treatment of acute spinal cord injury
Sella Pharmaceuticals, Inc.
As a diagnostic agent for the detection of growth hormone deficiency
CSL Behring
Treatment of patients needing urgent reversal of Vitamin K antagonist therapy for treatment of major bleeding and/or surgical procedures
Cleveland BioLabs, Inc & Incuron, LLC Joint Ventur
Treatment of hepatocellular carcinoma
ImClone Systems LLC
Treatment of gastric cancer
CSL Behring, LLC
Treatment of patients with congenital factor IX deficiency (hemophilia B).
Serendex ApS
Treatment of pulmonary alveolar proteinosis
Promedior, Inc.
Treatment of idiopathic pulmonary fibrosis.
Pfizer, Inc.
Treatment of Duchenne Muscular Dystrophy.
Ultragenyx Pharmaceutical, Inc.
Treatment of mucopolysaccharidosis VII (MPS VII, Sly Syndrome)
Jade Therapeutics LLC
Treatment of persistent corneal epithelial defects
Novartis Pharmaceuticals Corp.
Treatment of inclusion body myositis
Astellas Pharma Global Development, Inc.
Treatment of pancreatic cancer
Dompe S.p.A.
Prevention of graft loss in pancreatic islet transplantation
Onconova Therapeutics, Inc.
Treatment of ovarian cancer
Clovis Oncology, Inc.
Treatment of ovarian cancer
Orenova Group, LLC
Treatment of rare congenital ichthyoses.
The LAM Foundation
Treatment of lymphangioleiomyomatosis
Vascular Therapies, LLC
Prevention of arteriovenous(AV) fistula or AV graft failure in patients with end stage renal disease, receiving hemodialysis or preparing for hemodialysis
Stratatech Corporation
Treatment of hospitalized patients with complex skin defects resulting from partial and full thickness skin burns requiring excision and grafting
Hope Pharmaceuticals
Treatment of chlorine gas poisoning
Braintree Laboratories, Inc.
For cleansing of the colon in preparation for colonoscopic diagnosis of colonic disease in children and adolescents
Edinburg BioQuarter
Treatment of calciphylaxis
Medigen Biotechnology Corporation
Treatment of hepatocellular carcinoma
Alnylam Pharmaceuticals, Inc.
Treatment of familial amyloidotic polyneuropathy
Chiesi Pharmaceuticals, Inc.
Treatment of preterm neonatal respiratory distress syndrome
Eli Lilly and Company
Treatment of multiple myeloma
Lipella Pharmaceuticals Inc.
Treatment of hemorrhagic cystitis
Pfizer, Inc.
Treatment of symptomatic transthyretin (TTR) amyloid cardiomyopathy.
Stiefel Laboratories, Inc.
Treatment of congenital ichthyosis
Cerium Pharmaceuticals, Inc.
Treatment of infantile spasms
Debiopharm
Treatment of central precocious puberty
Genentech, Inc.
Treatment of pediatric patients (age 16 years and younger) with polyarticular-course juvenile idiopathic arthritis
Otsuka Pharmaceuticals Co., Ltd.
Treatment of autosomal dominant polycystic kidney disease
MedImmune Ltd.
Treatment of idiopathic pulmonary fibrosis
GlaxoSmithKline
Treatment of Stage IIb through IV melanoma.
Diffusion Pharmaceuticals, LLC
Treatment of brain metastasis
Lexicon Pharmaceuticals, Inc.
Management of symptoms of carcinoid syndrome associated with carcinoid tumor
Fera Pharmaceuticals, LLC
Treatment of endophthalmitis
Savara Pharmaceuticals, Inc.
Treatment of persistent methicillin-resistant S. aureus lung infection in patients with cystic fibrosis
Valeant Pharmaceuticals North America LLC
Treatment of chronic or recurrent central serous chorioretinopathy
Source: http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm
Threshold Pharmaceuticals, Inc.
1 Treatment of soft tissue sarcoma
Albireo AB
1 Treatment of progressive familial intrahepatic cholestatis
2 Treatment of primary biliary cirrhosis
Onconova Therapeutics, Inc.
1 Prevention of acute radiation toxicity, also known as Acute Radiation Syndrome (ARS)
2 Treatment of acute radiation symdrome
Atterocor, Inc.
Treatment of adrenocortical carcinoma
Xenon Pharmaceuticals, Inc.
Treatment of erythromelalgia
Del Mar Pharmaceuticals (BC), Ltd
Treatment of malignant gliomas
ViroDefense, Inc.
Treatment of poliovirus infection.
LifeSplice Pharma LLC
Treatment of amyotrophic lateral sclerosis
Millennium Pharmaceuticals, Inc
Treatment of systemic light chain (AL) amyloidosis.
Hoffmann-La Roche, Inc.
Treatment of Fragile X Syndrome
Can-Fite BioPharma Ltd.
Treatment of hepatocellular carcinoma
Cortendo AB (HQ address)
Treatment of endogenous Cushing's syndrome
AbbVie, Inc.
Treatment of chronic lymphocytic leukemia
Sarepta Therapeutics, Inc.
1 Prophylaxis for patients following documented or suspected exposure to ebolavirus
2 Prophylaxis following documented or suspected exposure to marburg virus
Immatics Biotechnologies GmbH
Treatment of renal cell carcinoma in HLA-A*2 positive patients
ReGenX Biosciences LLC
Treatment of homozygous familial hypercholesterolemia
Lonza
Treatment of hospitalized patients with deep partial and full thickness burns requiring grafting.
Dutch Opthalmic Research Cente
To selectively stain the thickened internal limiting membrane, which has formed onto the inner side of the retina in vitreo-retinal disorders
Coronado Biosciences, Inc.
Treatment of acute myeloid leukemia
Senesco Technologies, Inc.
1 Treatment of diffuse large B cell lymphoma
2 Treatment of mantle cell lymphoma
Unicorn Pacific Corporation
Treatment of multiple myeloma
Immuno Tech, Inc.
Treatment of Kaposi sarcoma
HanAll BioPharma Co., Ltd.
Treatment of Behcet's disease
Surgeon General of the U. S. Army
Prevention of hemorrhagic fever with renal syndrome caused by Hantaan virus and Puumala virus.
AndromedA Biotech, LTD
For use in type 1 diabetic mellitus patients with residual beta-cell function
Intercell AG
Prevention of Japanese encephalitis virus in pediatric patients.
ERYTECH Pharma
Treatment of pancreatic cancer.
CTI Science, Inc.
Treatment of mercury toxicity
Biokine Therapeutics Ltd.
For use in combination with G-CSF to mobilize HSCs from the marrow to peripheral blood for collection for autologous or allogeneic transplantation
Arno Therapeutics, Inc.
1 Treatment of Schwannoma of the central nervous system
2 Treatment of meningioma
Edward A. Neuwelt, MD
Prevention of ototoxicity caused by platinum-based chemotherapeutic agents used to treat pediatric cancers
JSK Therapeutics, Inc.
Treatment of acute myeloid leukemia
EcoPharm Corporation
Treatment of idiopathic thrombocytopenic purpura
PharmaIN Corporation
Treatment of refractory ascites due to all etiologies except for cancer
Apogenix GmbH
Treatment of Myelodysplastic Syndrome
Pfizer, Inc.
Routine prophylaxis to prevent bleeding episodes in patients with hemophilia A and B patients with inhibitors
AbbVie, Inc.
Treatment of glioblastoma multiforme (GBM).
Salupont Consulting Ltd
Treatment of severe pre-eclampsia
Rottapharm S.p.A.
Prevention of recurrent hepatitis C in liver transplant patients
Luitpold Pharmaceuticals, Inc.
Treatment of uremic and non-uremic calciphylaxis
Quark Pharmaceuticals, Inc.
Treatment of ischemic optic neuropathy
OncoTherapy Science, Inc.
Treatment of soft tissue sarcoma
Sigma-Tau Pharmaceuticals, Inc.
Treatment of Fragile X syndrome
Genable Technologies Limited / Smurfit Institute of Genetics
treatment of retinitis pigmentosa
Laboratorios del Dr. Esteve, S.A.
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Milo Biotechnology
Treatment of Duchennes and Becker's muscular dystrophy
Athersys, Inc.
Treatment of MPS-1, including Hurler syndrome
Boehringer Ingelheim Pharmaceuticals, Inc.
Treatment of epidermal growth factor receptor mutation positive non-small cell lung cancer
Niche Therapeutics, LLC
Treatment of ST-elevation myocardial infarcted patients who are undergoing primary percutaneous coronary intervention
Niche Therapeutics, LLC
Treatment of ST-elevation myocardial infarcted patients who are undergoing primary percutaneous coronary intervention
Millennium Pharmaceuticals, Inc.
Treatment of peripheral T-cell lymphoma
Neurotech USA, Inc.
Treatment of macular telangiectasia type 2 (MacTel)
Grifols Therapeutics, Inc.
Treatment of cystic fibrosis
Morphotek, Inc.
Treatment of mesothelioma
Recepta Biopharma S.A.
Treament of ovarian cancer
Bayer HealthCare Pharmaceuticals, Inc.
Treatment of mesothelioma
Isis Pharmaceuticals, Inc.
Treatment of familial amyloid polyneuropathy
LipimetiX Development, LLC
Treatment of homozygous familial hypercholesterolemia
bluebird bio, Inc.
Treatment of andrenoleukodystrophy
Duke University
Treatment of pediatric (0-16 yrs old inclusive) cerebral palsy due to acquired brain injury
Soligenix, Inc
Prevention of death following a potentially lethal dose of total body irradiation during or after a radiation disaster
Guochuan Emil Tsai, MD, PhD
University of California, Los Angeles
Treatment of pediatric schizophrenia
Gradalis, Inc.
Treatment of stage IIB to IV melanoma
Seattle Genetics, Inc.
Treatment of mycosis fungoides
Luitpold Pharmaceuticals, Inc.
Treatment of seizure prolongation in patients undergoing electroconvulsive therapy
Novartis Pharmaceuticals Corporation
Treatment of TNF-receptor associated periodic syndrome (TRAPS)
Orenova Group, LLC
Treatment of perforating diseases, including Kyrle's disease, reactive perforating collangenosis, perforating folliculitis and elastosis perforans serpiginosa
Hillhurst Biopharmaceuticals, Inc.
Treatment of sickle cell disease
SK Life Science, Inc.
Management of patients with infantile spasms
GANYMED Pharmaceuticals AG
Treatment of gastric cancer
Baxter Healthcare Corporation
Prophylactic use to prevent or reduce the frequency of bleeding episodes in patients with hemophilia B (routine prophylaxis in patients where there is no evidence or suspicion of bleeding)
OXiGene, Inc.
Treatment of acute myelogenous leukemia
Stephen G. Kaler, M.D.
Head, Unit on Human Copper Metabolism
Treatment of Menkes disease
AROG Pharmaceuticals, LLC
Treatment of acute myelogenous leukemia
Pfizer
1 Treatment of neuroblastoma
2 Treatment of anaplastic large cell lymphoma
Lumos Pharma
Treatment of creatine transporter deficiency
Eagle Pharmaceuticals, Inc.
Treatment of heat stroke
Luitpold Pharmaceuticals, Inc.
Treatment of trigeminal neuralgia
EryDel S.p.A.
Treatment of ataxia-telangiectasia
S. Cotter
Treatment of Prader Willi Syndrome
Acorda Therapeutics, Inc.
Management of patients with acute repetitive seizures
Glenveigh Medical, LLC
Treatment of severe preeclampsia and eclampsia
Proteo Biotech AG
Treatment of pulmonary arterial hypertension
Thrombologic
Treatment of purpura fulminans
Eisai, Inc.
Treatment of advanced soft tissue sarcoma
Novartis Pharmaceuticals Corporation
Treatment of hepatocellular carcinoma
Invenux, LLC
Treatment of sickle cell disease
ArsNova Partners, LLC
For use in magnetic resonance imaging to assess, and monitor treatment of solid tumor malignancies previously diagnosed in pediatric patients (age 16 years and younger)
XOMA (US) LLC
Treatment of non-infectious intermediate, posterior or pan uveitis, or chronic non-infectious anterior uveitis
Biodel, Inc.
Prevention of hypoglycemia in the congenital hyperinsulinism population
Cytheris, Inc.
Treatment of progressive multifocal leukoencephalopathy
Janssen Biotech, Inc.
1 Treatment of sarcoidosis
2 Treatment of pediatric ulcerative colitis
Cadila Pharmaceuticals Limited
Treatment of non-small cell lung cancers that express desmocollin-3
Promethera Biosciences
Treatment of urea cycle disorders
Promethera Biosciences
Treatment of Crigler-Najjar syndrome
Laboratoris Sanifit, S.L.
Treatment of calciphylaxis
Onclave Theraeputics Limited
Treatment of AA amyloidosis and AL amyloidosis
Galileo Research srl
Treatment of ovarian cancer
OCTAPHARMA USA, Inc.
Immune tolerance induction in hemophilia A patients with inhibitors
FibroGen, Inc.
Treatment of idiopathic pulmonary fibrosis
jCyte, Inc.
Treatment of retinitis pigmentosa
Merck, Sharp & Dohme Corp.
Treatment of Stage IIB through IV malignant melanoma
Pharmacyclics, Inc.
1 Treatment of chronic lymphocytic leukemia
2 Treatment of mantle cell lymphoma
3 Treatment of chronic lymphocytic leukemia (CLL).
Algorithum Sciences, LLC
Treatment of pulmonary arterial hypertension
AbbVie, Inc.
Treatment of ovarian cancer
Medgenics, Inc.
Treatment of chronic hepatitis D
Eisai, Inc.
Treatment of follicullar, medullary, anaplastic, and metastatic or locally advanced papillary thyroid cancer
Astellas Pharma Global Development, Inc.
Treatment of adrenocortical carcinoma
Aradigm Corporation
For the management of cystic fibrosis
REGiMMUNE Corporation
Prevention of graft-versus-host disease
Pharma Mar USA Inc.
Treatment of ovarian cancer
PCUT BioPartners, Inc.
Treatment of amyotropic lateral sclerosis
Premacure AB
Prevention of retinopathy of prematurity in premature infants born at risk for the disease
Icon Bioscience, Inc.
Treatment of retinoblastoma
OncoTx, LLC
Treatment of Stage IIB through IV melanoma
Nu Science Trading, LLC
Treatment of calciphylaxis
Nova Laboratories Limited
Treatment of acute lymphoblastic leukemia in pediatric patients
Fera Pharmaceuticals, LLC
Treatment of methemoglobinemia
Luitpold Pharmaceuticals, Inc.
Treatment of congenital and acquired methemoglobinemia
Provepharm SAS
Treatment of hereditary methemoglobinemia
Laboratoire HRA Pharma
Treatment of Cushing's syndrome
Meridian Medical Technologies, Inc.
Treatment of nerve agent-induced seizures
Nerviano Medical Sciences S.r.l.
Treatment of thymic epithelial tumors.
Gencia Corporation
Treatment of inherited mitochondrial respiratory chain disease
APEIRON Biologics AG
Treatment of neuroblastoma.
Luitpold Pharmaceuticals, Inc.
Treatment of Myasthenia Gravis
GeNO, LLC
Treatment of persistent pulmonary hypertension in newborns
Genentech, Inc
1 Treatment of chronic lymphocytic leukemia
2 Treatment of diffuse large B cell lymphoma
Aerial BioPharma, LLC
Treatment of narcolepsy
Terra Biological LLC
Treatment of gliomas
Cell Therapeutics, Inc.
Treatment of glioblastoma multiforme
Novartis Pharmaceuticals Corporation
Treatment of multiple myeloma
PharmaEssentia Corporation
Treatment of polycythemia vera
Keck Graduate Institute of Applied Life Sciences
Treatment of Behcet's disease
Eisai, Inc.
Treatment of Lennox-Gastaut Syndrome
Heart Metabolics, Ltd.
Treatment of moderate to severe symptomatic (NYHA class III or IV) hypertrophic cardiomyopathy
Luitpold Pharmaceuticals, Inc.
Treatment of Tetralogy of Fallot
TRB Chemedica International S.A.
Treatment of acute spinal cord injury
Sella Pharmaceuticals, Inc.
As a diagnostic agent for the detection of growth hormone deficiency
CSL Behring
Treatment of patients needing urgent reversal of Vitamin K antagonist therapy for treatment of major bleeding and/or surgical procedures
Cleveland BioLabs, Inc & Incuron, LLC Joint Ventur
Treatment of hepatocellular carcinoma
ImClone Systems LLC
Treatment of gastric cancer
CSL Behring, LLC
Treatment of patients with congenital factor IX deficiency (hemophilia B).
Serendex ApS
Treatment of pulmonary alveolar proteinosis
Promedior, Inc.
Treatment of idiopathic pulmonary fibrosis.
Pfizer, Inc.
Treatment of Duchenne Muscular Dystrophy.
Ultragenyx Pharmaceutical, Inc.
Treatment of mucopolysaccharidosis VII (MPS VII, Sly Syndrome)
Jade Therapeutics LLC
Treatment of persistent corneal epithelial defects
Novartis Pharmaceuticals Corp.
Treatment of inclusion body myositis
Astellas Pharma Global Development, Inc.
Treatment of pancreatic cancer
Dompe S.p.A.
Prevention of graft loss in pancreatic islet transplantation
Onconova Therapeutics, Inc.
Treatment of ovarian cancer
Clovis Oncology, Inc.
Treatment of ovarian cancer
Orenova Group, LLC
Treatment of rare congenital ichthyoses.
The LAM Foundation
Treatment of lymphangioleiomyomatosis
Vascular Therapies, LLC
Prevention of arteriovenous(AV) fistula or AV graft failure in patients with end stage renal disease, receiving hemodialysis or preparing for hemodialysis
Stratatech Corporation
Treatment of hospitalized patients with complex skin defects resulting from partial and full thickness skin burns requiring excision and grafting
Hope Pharmaceuticals
Treatment of chlorine gas poisoning
Braintree Laboratories, Inc.
For cleansing of the colon in preparation for colonoscopic diagnosis of colonic disease in children and adolescents
Edinburg BioQuarter
Treatment of calciphylaxis
Medigen Biotechnology Corporation
Treatment of hepatocellular carcinoma
Alnylam Pharmaceuticals, Inc.
Treatment of familial amyloidotic polyneuropathy
Chiesi Pharmaceuticals, Inc.
Treatment of preterm neonatal respiratory distress syndrome
Eli Lilly and Company
Treatment of multiple myeloma
Lipella Pharmaceuticals Inc.
Treatment of hemorrhagic cystitis
Pfizer, Inc.
Treatment of symptomatic transthyretin (TTR) amyloid cardiomyopathy.
Stiefel Laboratories, Inc.
Treatment of congenital ichthyosis
Cerium Pharmaceuticals, Inc.
Treatment of infantile spasms
Debiopharm
Treatment of central precocious puberty
Genentech, Inc.
Treatment of pediatric patients (age 16 years and younger) with polyarticular-course juvenile idiopathic arthritis
Otsuka Pharmaceuticals Co., Ltd.
Treatment of autosomal dominant polycystic kidney disease
MedImmune Ltd.
Treatment of idiopathic pulmonary fibrosis
GlaxoSmithKline
Treatment of Stage IIb through IV melanoma.
Diffusion Pharmaceuticals, LLC
Treatment of brain metastasis
Lexicon Pharmaceuticals, Inc.
Management of symptoms of carcinoid syndrome associated with carcinoid tumor
Fera Pharmaceuticals, LLC
Treatment of endophthalmitis
Savara Pharmaceuticals, Inc.
Treatment of persistent methicillin-resistant S. aureus lung infection in patients with cystic fibrosis
Valeant Pharmaceuticals North America LLC
Treatment of chronic or recurrent central serous chorioretinopathy
Source: http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm
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