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
(S)-3-((S)-2-(2-((2,6-difluorophenyl)amino)-2-oxoacetamido)propanamido)-4-oxo-5-(2,3,5,6-tetrafluorophenoxy)pentanoic acid Primary sclerosing cholangitis Pharma Gateway AB October 16, 2017
Pracinostat Acute myeloid leukaemia Helsinn Birex Pharmaceuticals Ltd October 16, 2017
2-[N-(2-hydroxyethyl)]-N-(4-methoxybenzenesulfonyl)]amino-N-(4-chlorocinnamyl)-N-methylbenzylamine Charcot-Marie-Tooth disease Repositioning SAS October 16, 2017
Recombinant adeno-associated viral vector serotype 5 encodingStaphylococcus aureusCas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of theCEP290gene Leber congenital amaurosis Pharma Gateway AB October 16, 2017
5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate] Biliary tract cancer Voisin Consulting S.A.R.L. October 16, 2017
Recombinant monoclonal antibody to sialic acid-binding Ig-like lectin 8 Mastocytosis Envestia Limited October 16, 2017
Adenoviral vector of serotype 5 modified to contain a chimeric sequence consisting of a minimal urokinase-type plasminogen activator receptor promoter preceded by three Notch-responsive elements, and coated with oligopeptide end-modified poly (beta-amino) esters Pancreatic Cancer Sagetis Biotech, S.L. October 16, 2017
Seladelpar Primary biliary cirrhosis Larode Ltd October 16, 2017
Autologous ex-vivo-expanded peripheral polyclonal lymphocytes enriched in activated natural killer cells Multiple myeloma CellProtect Nordic Pharmaceuticals AB October 16, 2017
Siplizumab Treatment in solid organ transplantation ITB-MED AB October 16, 2017
Bitopertin Beta-thalassaemia intermedia and major Roche Registration Limited October 16, 2017
Synthetic cyclic 8 amino acid analogue of human unacylated ghrelin Prader-Willi syndrome Alizé Pharma October 16, 2017
Synthetic cyclic 8 amino acid analog of human unacylated ghrelin Prader-Willi syndrome Aliz� Pharma;15G Chemin du Saquin;�cully October 12, 2017
Autologous glioma tumor cells treated with 18-mer oligodeoxynucleotide sequence complementary to insulin-like growth factor type 1 receptor (IGF-1R) codons 2-7 in the IG-1R gene Glioma Thomas Jefferson University;Department of Neurological Surgery, 909 Walnut Street;Philadelphia, Pennsylvania, 19107 October 11, 2017
Avelumab Stomach Cancer EMD Serono Research & Development Institute, Inc.;45A Middlesex Turnpike;Billerica, Massachusetts, 01821 October 11, 2017
(2-(3-(4-(1H-indazol-5-ylamino)quinazolin-2-yl)phenoxy)-N-isopropylacetamide-methane sulfonic acid salt) Graft Versus Host Disease Kadmon Corporation, LLC;450 East 29th Street;New York, New York, 10016 October 5, 2017
Autologous T lymphocyte enriched population cultured in the rpesence of phosphatidylinositol 3-kinase inhibitor (bb007) that contains cells transduced with an anti-human B cell maturation antigen (BCMA02) chimeric antigen receptor (CAR) lentiviral vector encoding a CAR that targets human BCMA Multiple myeloma bluebird bio, Inc.;60 Binney Street;Cambridge, Massachusetts, 02142 October 5, 2017
Filgotinib maleate Ulcerative colitis Gilead Sciences, Inc.;199 E. Blaine St.;Seattle, Washington, 98102 October 5, 2017
Pembrolizumab Small Cell Lung Cancer excluding those that have been identified as having a biomarker referred to as microsatellite instability-high (MSI-H) or mismatched repair deficient (dMMR) Merck, Sharp, & Dohme, a subsidiary of Merck & Co., Inc.;1 Merck Drive;Whitehouse Station, New Jersey, 08889 October 3, 2017
6-{[(1R,2S)-2-aminocyclohexyl]amino}-7-fluoro-4-(1-methyl-1H-pyrazol-4-yl)-1,2-dihydro-3H-pyrrolo[3,4-c]pyridin-3-one monocitrate Acute myeloid leukemia Millennium Pharmaceuticals, Inc.;40 Landsdowne Street;Cambridge, Massachusetts, 02139 October 3, 2017
Adeno Associated Virus carried Multi Characteristic Opsin Retinitis pigmentosa NanoScope Technologies;1312 Brown Trail;Bedford, Texas, 76022 October 3, 2017
Gaboxadol Fragile X syndrome Ovid Therapeutics;1460 Broadway;New York, New York, 10036 October 3, 2017
Recombinant humanized immunoglobulin gamma 1 monoclonal antibody (mAb) directed against the CemX segment of human membrane-bound immunoglobulin E (mIgE) Hyper-IgE Syndrome Fountain Biopharma, Inc.;19F-1, No. 3, 3 Yuan Qu Street;Nangang District September 28, 2017
Tazemetostat Malignant pleural mesothelioma Epizyme Inc.;400 Technology Square, 4th Floor;Cambridge, Massachusetts, 02139 September 28, 2017
1-(2-hydroxyethyl)-8-{[5-(4-methylpiperazin-1-yl)-2-(trifluoromethoxy) phenyl]amino}-4,5-dihydro-1H-pyrazolo[4,3-h]quinazoline-3-carboxamide fumarate salt Acute myeloid leukemia Trovagene, Inc.;11055 Flintkote Avenue;San Diego, California, 92121 September 28, 2017