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
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
Autologous human peripheral blood mononuclear cells incubated ex vivo with protein TBX-4000 (Tat-MYC) Malignant melanoma Stages IIb to IV Taiga Biotechnologies, Inc.;12635 Montview Boulevard, Fitzsimmons BBioscience Park Center;Aurora, Colorado, 80045 October 16, 2017
Entospletinib Acute myeloid leukaemia Gilead Sciences International Ltd. October 16, 2017
Seladelpar Primary biliary cirrhosis Larode Ltd October 16, 2017
Glasdegib maleate Acute myeloid leukaemia Pfizer Limited October 16, 2017
Synthetic cyclic 8 amino acid analogue of human unacylated ghrelin Prader-Willi syndrome Alizé Pharma October 16, 2017
Pracinostat Acute myeloid leukaemia Helsinn Birex Pharmaceuticals Ltd October 16, 2017
Recombinant monoclonal antibody to sialic acid-binding Ig-like lectin 8 Mastocytosis Envestia Limited October 16, 2017
Siplizumab Treatment in solid organ transplantation ITB-MED AB October 16, 2017
(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
2-[N-(2-hydroxyethyl)]-N-(4-methoxybenzenesulfonyl)]amino-N-(4-chlorocinnamyl)-N-methylbenzylamine Charcot-Marie-Tooth disease Repositioning SAS 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
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
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
(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
Gaboxadol Fragile X syndrome Ovid Therapeutics;1460 Broadway;New York, New York, 10036 October 3, 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
Autologous CD4+/CD8+ T cells expressing a BCMA-specific chimeric antigen receptor (CAR) Multiple myeloma Juno Therapeutics, Inc.;400 Dexter Avenue North, Suite 1200;Seattle, Washington, 98109 September 28, 2017
Givinostat Polycythemia vera Italfarmaco SpA;54 Via dei Lavoratori;Cinisello Balsamo September 28, 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