Treatments

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

You can filter the content by clicking on the titles: Product, Indication, Sponsor and Date. Clicking once, will filter the column alphabetically from A – Z and a second click on the same title will filter from Z – A.

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Country Product Indication Sponsor Date
Lenalidomide Extranodal marginal zone lymphoma of mucosa-associated lymphoid tissue Celgene Corporation;400 Connell Drive, Suite 7000;Berkley Heights, NJ, 07922 April 29, 2015
Lenalidomide Extranodal marginal zone lymphoma of mucosa-associated lymphoid tissue Celgene Corporation;86 Morris Avenue;Summit, New Jersey, 07901 April 29, 2015
(S)-7-(1-(9H-purin-6-ylamino)ethyl)-6-(3-fluorophenyl)-3-methyl-5H-thiazolo[3,2-a]pyrimidin-5-one Hodgkin’s disease Incyte Corporation;1801 Augustine Cut-Off;Wilmington, Delaware, 19803 April 29, 2015
Propranolol and etodolac Pancreatic cancer Vicus Therapeutics, LLC;55 Madison Avenue, Suite 400;Morristown, New Jersey, 07960 April 27, 2015
Docosahexaenoic acid Sickle cell anemia Sancilio & Company, Inc.;3874 Fiscal Ct., #100;Riviera Beach, Florida, 33404 April 27, 2015
Liposomal P-ethoxy growth factor receptor-bound protein 2 antisense oligonucleotide Acute myeloid leukemia Bio-Path Holdings, Inc.;4710 Bellaire Blvd, Suite 210;Houston, Texas, 77401 April 27, 2015
Phenol, 4-[2-(aminomethyl)-4-thiazolyl]-2,6-bis(1,1-dimethylethyl)monohydrochloride Huntington disease Ipsen Pharma April 24, 2015
Adeno-associated viral vector serotype 5 containing the humanCHMgene Choroideremia Inserm-Transfert SA April 24, 2015
Lenalidomide Marginal zone lymphoma Celgene Europe Limited April 24, 2015
Fluciclovine (18F) Glioma Blue Earth Diagnostics Ltd April 24, 2015
Sodium 2-hydroxylinoleate Neuroblastoma Ability Pharmaceuticals SL April 24, 2015
Xenon Perinatal asphyxia Neuroprotexeon Ltd April 24, 2015
Ecothiopate iodide Stargardt's disease Dorian Regulatory Affairs BV April 24, 2015
1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride Tularaemia Arno Therapeutics UK, Limited April 24, 2015
Human reovirus type 3 Dearing strain Pancreatic Cancer Oncolytics Biotech (UK) Limited April 24, 2015
Recombinant monoclonal IgG1 antibody against T-cell immune response cDNA 7 Prevention of graft rejection following solid organ transplantation Nekonal S.a.r.l. April 24, 2015
Recombinant human mesencephalic astrocyte-derived neurotrophic factor Retinitis pigmentosa Regintel Limited April 24, 2015
Ecothiophate iodide Stargardt's disease JJGConsultancy Ltd April 24, 2015
1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride Cryptococcosis Arno Therapeutics UK, Limited April 24, 2015
Rimeporide Duchenne muscular dystrophy EUDRAC Limited April 24, 2015
Rintatolimod Ebola NV Hemispherx BioPharma Europe April 24, 2015
Recombinant human histidyl tRNA synthetase Facioscapulohumeral muscular dystrophy aTyr Pharma, Inc.;3545 John Hopkins Court, Suite 250;San Diego, California, 92121 April 22, 2015
Synthetic double-stranded (hybridized duplex) ribonucleic acid oligonucleotide specific to hydroxyacid oxidase 1 gene Primary Hyperoxaluria Type 1 Dicerna Pharmaceuticals, Inc.;87 Cambridgepark Drive;Cambridge, Massachusetts, 02140 April 22, 2015
Cannabidiol Neonatal hypoxic ischemic encephalopathy GW Pharma Ltd.;15 T.W. Alexander Drive, P. O. Box 13547;Research Triangle Park, North Carolina, 27709 April 22, 2015
Allogeneic cardiosphere-derived cells Duchenne muscular dystrophy Capricor, Inc.;8840 Wilshire Blvd., 2nd Floor;Beverly HIlls, California, 90211 April 21, 2015