Treatments

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

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Country Product Indication Sponsor Date
Cabozantinib Hepatocellular Carcinoma Exelixis, Inc.;210 East Grand Avenue;South San Francisco, California, 94083 March 2, 2017
Melatonin Acute acetaminophen overdose Therapicon SrL;21 Via Malachia Marchesi de Taddei;Milano March 2, 2017
Hydroxyurea Sickle cell anemia Nova Laboratories Limited;Martin House, Gloucester Crescent;Leicester March 2, 2017
3-pentylbenzeneacetic acid sodium salt Alström syndrome ProMetic BioTherapeutics, Inc;1330 Piccard Drive, Suite 201;Rockville, Maryland, 20850 February 28, 2017
(s)-perillyl alcohol temozolomide carbamate Intracranially located metastases from breast cancer NeOnc Technologies, Inc.;8335 Sunset Blvd., #240;Los Angeles, California, 90069 February 28, 2017
3-pentylbenzeneacetic acid sodium salt Alstr�m syndrome ProMetic BioTherapeutics, Inc;1330 Piccard Drive, Suite 201;Rockville, Maryland, 20850 February 28, 2017
Tauroursodeoxycholic acid Amyotrophic lateral sclerosis Bruschettini s.r.l. February 27, 2017
Adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and the human iduronate 2-sulfatase (hIDS) gene Hunter Syndrome Sangamo Therapeutics, Inc.;Point Richmond Tech Center, 501 Canal Boulevard;Richmond, California, 94804 February 27, 2017
1-(2,2-difluoro-2H-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropane-1-carboxamide and ivacaftor Cystic fibrosis Vertex Pharmaceuticals (Europe) Limited February 27, 2017
26 base synthetic single-stranded fully phosphorothioated 2′-O-methyl-RNA and DNA mixmer oligonucleotide-based compound Dravet Syndrome Eirgen Pharma Limited February 27, 2017
Alpha-tocopherol and ascorbic acid Fragile X syndrome Advanced Medical Projects February 27, 2017
5-(4,6-dimorpholino-1,3,5-triazin-2-yl)-4-(trifluoromethyl)pyridin-2-amine Diffuse Large B-cell Lymphoma Voisin Consulting S.A.R.L. February 27, 2017
Autologous T cells transduced with lentiviral vector encoding an anti-SLAMF7 CD28/CD3-zeta chimeric antigen receptor Multiple myeloma Dr. Michael Hudecek February 27, 2017
505 amino acid protein, corresponding to amino acids 2-506 of the wild-type human histidyl-tRNA synthetase Limb-girdle muscular dystrophy Voisin Consulting S.A.R.L. February 27, 2017
Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-L-glutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl]acetate salt Primary ciliary dyskinesia Polyphor UK Ltd February 27, 2017
Fenfluramine hydrochloride Lennox-Gastaut syndrome Zogenix International Ltd February 27, 2017
Asciminib Chronic myeloid leukemia Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 February 27, 2017
Ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing theCOL7A1gene Dystrophic epidermolysis bullosa Abeona Therapeutics Europe SL February 27, 2017
Humanised IgG4 monoclonal antibody to the human toll-like receptor type 2 Pancreatic Cancer Opsona Therapeutics Limited February 27, 2017
Humanised IgG4 monoclonal antibody to the human toll-like receptor type 2 Myelodysplastic syndromes Opsona Therapeutics Limited February 27, 2017
Propranolol hydrochloride Von Hippel-Lindau syndrome Consejo Superior de Investigaciones Cientificas (CSIC) February 27, 2017
Fully human IgG1 antibody specific for CD33 Myelodysplastic syndromes Boehringer Ingelheim Pharmaceuticals, Inc.;900 Ridgebury Rd., PO Box 368;Ridgefield, Connecticut, 06877 February 27, 2017
Iodine (131I) murine IgG1 monoclonal antibody against CD276 Neuroblastoma Y-mAbs Therapeutics A/S February 27, 2017
Recombinant human club cell 10 KDa protein Bronchiolitis obliterans syndrome EUDRAC Limited February 27, 2017
N-(4-(1-cyanocyclopentyl)phenyl)-2-(4-pyridinylmethyl)amino-3-pyridinecarboxamide methanesulfonate Stomach Cancer Sirius Regulatory Consulting Limited February 27, 2017