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
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
Fenfluramine hydrochloride Lennox-Gastaut syndrome Zogenix International Ltd February 27, 2017
Soluble recombinant human fibroblast growth factor receptor 3 Achondroplasia TherAchon SAS February 27, 2017
Humanised IgG4 monoclonal antibody to the human toll-like receptor type 2 Pancreatic Cancer Opsona Therapeutics Limited February 27, 2017
Tauroursodeoxycholic acid Amyotrophic lateral sclerosis Bruschettini s.r.l. February 27, 2017
Propranolol hydrochloride Von Hippel-Lindau syndrome Consejo Superior de Investigaciones Cientificas (CSIC) February 27, 2017
Recombinant human club cell 10 KDa protein Bronchiolitis obliterans syndrome EUDRAC Limited February 27, 2017
Thalidomide Hereditary haemorrhagic telangiectasia PlumeStars s.r.l. February 27, 2017
Vemurafenib Erdheim-Chester syndrome Groupe d'étude des histiocytoses 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
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
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
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 Myelodysplastic syndromes Opsona Therapeutics Limited 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
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