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
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
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
Autologous ex-vivo-expanded peripheral polyclonal lymphocytes enriched in activated natural killer cells Multiple myeloma CellProtect Nordic Pharmaceuticals AB October 16, 2017
Cannabidiol West syndrome GW Research Ltd October 16, 2017
Upamostat Pancreatic Cancer RedHill Biopharma Ltd.;21 Ha'arba'a Street;Tel Aviv-Yafo October 16, 2017
Bitopertin Beta-thalassaemia intermedia and major Roche Registration Limited October 16, 2017
Cannabidivarin Rett syndrome GW Research Ltd October 16, 2017
Glucopyranosyl lipid A Follicular lymphoma Immune Design Ltd October 16, 2017
Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F Multiple myeloma GlaxoSmithKline Trading Services Limited October 16, 2017
Ofranergene obadenovec Ovarian cancer Envigo Pharma Consulting Ltd 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
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
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