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
Fixed-dose combination of fosfomycin disodium and tobramycin Cystic fibrosis CURx Pharma (UK) Limited August 10, 2015
Insulin human Short bowel syndrome Sirius Regulatory Consulting Limited August 10, 2015
Adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene Hunter Syndrome Laboratorios del Dr Esteve, S.A. August 10, 2015
Fibrinogen-coated albumin spheres Acute Radiation Syndrome Acute radiation syndrome Fibreu Limited August 10, 2015
Ibrutinib Marginal zone lymphoma Janssen-Cilag International NV August 10, 2015
Adeno-associated viral vector serotype 8 containing the humanMTM1gene Alport syndrome Audentes Therapeutics UK Limited August 10, 2015
2-(2-phenylvinyl)-4-[4-methylpiperazin-1-yl)]-6-(5-methyl-2H-pyrazol-3-yl-amino)-pyrimidine L(+) tartrate salt Hepatocellular Carcinoma Dr Ulrich Granzer August 10, 2015
(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes Khondrion BV August 10, 2015
Lanreotide acetate Polycystic kidney disease- type 1 Prof. Dr R.T.Gansevoort August 10, 2015
CD33-directed antibody-drug conjugate consisting of an antibody conjugated to a DNA cross-linking pyrrolobenzodiazepine dimer drug Acute myeloid leukaemia Seattle Genetics UK, Limited August 10, 2015
Daratumumab Follicular lymphoma Janssen Research & Development, LLC;1400 McKean Road, P. O. Box 776;Spring House, Pennsylvania, 19477 August 6, 2015
Butylidenephthalide Glioma Everfront Biotech, Inc.;11F., No. 31, Ln. 169, Kangning St., New Taipei City 221; August 5, 2015
Sodium valproate Wolfram syndrome The University of Birmingham;Birmingham Research Park - Vincent Drive, Birmingham B15 2SQ; August 5, 2015
Varlitinib Bile duct cancer ASLAN Pharmaceuticals;10A Bukit Pasoh Road, 089824; August 5, 2015
Inecalcitol Acute myeloid leukemia Hybrigenics, S.A.;3-5 Impasse Reille, 75014;Paris August 3, 2015
Afatinib Non-small cell lung cancer Boehringer Ingelheim Pharmaceuticals, Inc.;900 Ridgebury Road, P. O. Box 368;Ridgefield, Connecticut, 06877 August 3, 2015
2-((3-((4-((3-aminopropyl)amino)butyl)amino)propyl)amino)-N-((5S,5aS,8aR,9R)-9-(4-hydroxy-3,5-dimethoxyphenyl)-8-oxo-5,5a,6,8,8a,9-hexahydrofuro[3′,4′:6,7]naphtho[2,3-d][1,3]dioxol-5-yl)acetamide, tetrahydrochloride Acute myeloid leukaemia Pierre Fabre Médicament July 28, 2015
Sarizotan hydrochloride Rett syndrome Newron Pharmaceuticals SpA July 28, 2015
Adenovirus-associated viral vector serotype 2 containing the humanRPE65gene Retinitis pigmentosa Spark Therapeutics Ireland Ltd July 28, 2015
Anti-H5N1 equine immunoglobulin F(ab’)2fragments Avian influenza Fab'entech July 28, 2015
Veltuzumab Immune thrombocytopenic purpura Immunomedics, Inc.;300 The American Road;Morris Plains, New Jersey, 07950 July 28, 2015
Inecalcitol Acute myeloid leukaemia Hybrigenics SA July 28, 2015
Beloranib Craniopharyngioma FGK Representative Service GmbH July 28, 2015
Triheptanoin Long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency Ultragenyx UK Limited July 28, 2015
Triheptanoin Carnitine palmitoyltransferase II deficiency Ultragenyx UK Limited July 28, 2015