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
Soraprazan Stargardt Disease Katairo GmbH;21 Lederstra�e;Kusterdingen December 22, 2017
2-O, 3-O, desulfated heparin Acute myeloid leukemia Cantex Pharmaceuticals, Inc.;1792 Bell Tower Lane;Weston, Florida, 33326 December 21, 2017
Rapamycin Perivascular epithelioid cell tumors (PEComa) Aadi Bioscience, Inc.;1343 Luna Vista Drive;Los Angeles, California, 90272 December 21, 2017
Methyl (2R,3S)-3-[(methylsulfonyl)amino]-2-{[(cis-4-phenylcyclohexyl)oxy]methyl}piperidine-1-carboxylate Narcolepsy Takeda Development Center Americas, Inc.;Takeda Parkway;Deerfield, Illinois, 60015 December 19, 2017
Mosedipimod Acute Radiation Syndrome Acute radiation syndrome Enzychem Lifesciences Corporation;59, Bio valley-ro;Jecheon-si December 19, 2017
Sodium sulfate, magnesium sulfate, potassium chloride For the cleansing of the colon in preparation for colonoscopic diagnosis of colonic disease in pediatric patients Braintree Laboratories, Inc.;60 Columbian Street West, P.O. Box 850929;Braintree, Massachusetts, 02185 December 19, 2017
Fenretinide Cutaneous T-cell lymphoma SciTech Development, LLC;281 Kercheval Avenue;Grosse Pointe Farms, Michigan, 48236 December 19, 2017
(S)-2-(1-(9H-purin-6-ylamino)propyl)-3-(3-fluorophenyl)-4H-chromen-4-one) T-cell lymphoma Rhizen Pharmaceuticals SA;40 Rue Fritz-Courvoisier;La Chaux-de-Fonds December 18, 2017
Diacetyl(N(4)-methylthiosemicarbazonato) copper (II) Amyotrophic lateral sclerosis Collaborative Medicinal Development Pty. Ltd. (CMD);c/o Shaw Downie, Level 11;Sydney December 18, 2017
Lentiviral transfected chimeric antigen receptor T-cells modified to target B-cell maturation antigen and CS1 Multiple myeloma iCell Gene Therapeutics;25 Health Sciences Drive, Suite 118;Stony Brook, New York, 11793 December 14, 2017
Plasminogen (Human) Idiopathic pulmonary fibrosis ProMetic BioTherapeutics, Inc;1330 Piccard Drive, Suite 201;Rockville, Maryland, 20850 December 14, 2017
Metformin Progressive myoclonus epilepsy type 2 (Lafora disease) Consorcio Centro de Investigaci�n Biom�dica en Red, M.P. (CIBER);Monforte de Lemos, 3-5 Pabellon 11;Madrid December 14, 2017
Dual signaling protein fusing cytotoxic T-lymphocyte-associated protein 4 (CTLA-4) to the Fas-ligand (FasL) Diffuse Large B-cell Lymphoma KAHR Medical, LTD;P. O. Box 12000;Jerusalem December 13, 2017
Adenovirus associated viral vector serotype 8 containing the humanAIPL1gene Leber congenital amaurosis MeiraGTx UK II Limited December 12, 2017
Agammaglobulinaemia tyrosine kinase Pemphigus Clinical Network Services (UK) Ltd December 12, 2017
Venetoclax Mantle cell lymphoma AbbVie Ltd December 12, 2017
(2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamide Paroxysmal nocturnal hemoglobinuria FGK Representative Service GmbH December 12, 2017
2-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione Mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes NeuroVive Pharmaceutical AB December 12, 2017
4-hydroxy-2,2,6,6-tetramethylpiperidine-N-oxyl Familial cerebral cavernous malformations Premier Research Group Limited December 12, 2017
Acetylleucine GM2 gangliosidosis IntraBio Ltd December 12, 2017
Modified messenger ribonucleic acid encoding human argininosuccinate lyase enzyme encapsulated into lipid nanoparticles Argininosuccinic Aciduria PhaseRx Ireland, Ltd December 12, 2017
Pegunigalsidase alfa Fabry disease Protalix B.V December 12, 2017
Cholesterol 24S-hydroxylase inhibitor Lennox-Gastaut syndrome Takeda Development Center Americas, Inc.;Takeda Parkway;Deerfield, Illinois, 60015 December 6, 2017
D-amino acid oxidase inhibitor Friedreich Ataxia Takeda Development Center Americas, Inc.;Takeda Parkway;Deerfield, Illinois, 60015 December 6, 2017
Gemfibrozil and vitamin A Neuronal ceroid lipofuscinoses Polaryx Therapeutics, Inc.;140 East Ridgewood Avenue, Suite 415, South Tower;Paramus, New Jersey, 07652 December 6, 2017