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
N-(4-Methoxyphenyl)-N,2,6-trimethylfuro[2,3-d] pyrimidin-4-amine Glioma Flag Therapeutics, Inc.;9168 Wooden Road;Raleigh, North Carolina, 27617 February 18, 2016
Methyl 3-((2R)-2-hydroxy-4-(((((S)-1-methoxy-1-oxopropan-2-yl) amino)(phenoxy)phosphoryl)oxy)-3,3-dimethylbutanamido)propanoate Pantothenate-kinase-associated neurodegeneration Retrophin Europe Limited February 17, 2016
Humanised IgG4 monoclonal antibody against total complement component 1, subcomponent s Autoimmune hemolytic anemia Assign Group Development UK Ltd February 17, 2016
Tolfenamic acid Frontotemporal dementia RV Developpement February 17, 2016
Pembrolizumab Multiple myeloma Merck, Sharp & Dohme Corp.;One Merck Drive;Whitehouse Station, New Jersey, 08889 February 17, 2016
Ex-vivo-expanded autologous fibroblasts transduced with lentiviral vector containing theCOL7A1gene Dystrophic epidermolysis bullosa Dr Waseem Qasim February 17, 2016
Diclofenamide Periodic paralysis Prof. Michael Hanna February 17, 2016
Tolfenamic acid Progressive supranuclear palsy RV Developpement February 17, 2016
Venetoclax Acute myeloid leukaemia AbbVie Ltd February 17, 2016
Dantrolene sodium Wolfram syndrome Washington University in St. Louis;660 S. Euclid Avenue, Campus Box 8127;St. Louis, Missouri, 63110 February 17, 2016
2-ethylbutyl (2S)-2-{[(S)-{[(2R,3S,4R,5R)-5-(4-aminopyrrolo[2,1-f][1,2,4]triazin-7-yl)-5-cyano-3,4-dihydroxytetrahydrofuran-2-yl]methoxy}(phenoxy)phosphoryl]amino}propanoate Ebola Gilead Sciences International Ltd. February 17, 2016
Allogeneic fetal human retinal progenitor cells expanded ex vivo Retinitis pigmentosa Voisin Consulting S.A.R.L. February 17, 2016
N-(4-methoxyphenyl)-N,2,6-trimethylfuro[2,3-d]pyrimidin-4-amine Glioma FLAG Therapeutics Ltd February 17, 2016
Delta-9-tetrahydrocannabinol and cannabidiol from extracts of theCannabis sativaL. plant Glioma GW Research Ltd February 17, 2016
DNA plasmid encoding a recombinant fusion protein consisting of the extracellular domain of human TNFa p55 receptor linked to the human IgG1 Fc domain Non-infectious uveitis Eyevensys SAS February 17, 2016
S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-N-methyl-L-isoleucinamide) C3 glomerulopathy Amyndas Pharmaceuticals S.A. February 17, 2016
Arsenic trioxide Acute myeloid leukaemia Orsenix Holdings BV February 17, 2016
1E,6E)-1,7-Bis(3,4-dimethoxyphenyl)-4-cyclobutylmethyl-1,6-heptadiene-3,5-dione or [(1E, 4Z,6E)-4-(cyclobutylmethyl)-1,7-bis(3,4-dimethoxyphenyl)-5-hydroxyhepta-1,4,6-trien-3-one] Spinal muscular atrophy Allianz Pharmascience Ltd.;8630 Fenton Street, Suite 724;Silver Spring, Maryland, 20910 February 17, 2016
6-{4-[l-(propan-2-yl)piperidin-4-yl]-1,4-diazepan-1-yl}-N-(pyridin-4-yl)pyridine-2-carboxamide Glioblastoma Proximagen, LLC;505 Waterford Park, Hwy 169 North;Plymouth, Minnesota, 55441 February 11, 2016
Dodecafluoropentane emulsion Sickle cell anemia NuvOx Pharma;1635 East 18th Street;Tucson, Arizona, 85719 February 11, 2016
Epacadostat Stage IIB-IV melanoma Incyte Corporation;1801 Augustine Cut-Off;Wilmington, Delaware, 19803 February 11, 2016
Humanized affinity-optimized, afucosylated immunoglobulin G1 kappa monoclonal antibody against CD19 Neuromyelitis optica MedImmune, LLC;1 Medimmune Way;Gaithersburg, Maryland, 20878 February 10, 2016
Midazolam Seizures induced by organophosporous insecticide poisoning Meridian Medical Technologies, Inc.;A Pfizer Company, 6350 Stevens Forest Road;Columbia, Maryland, 21046 February 8, 2016
Rezafungin Candidemia and invasive candidiasis infections caused by susceptible Candida spp Cidara Therapeutics, Inc.;6310 Nancy Ridge Drive, Suite 101;San Diego, California, 92121 February 8, 2016
Angiotensin (1-7) LAMA2-related muscular dystrophy Tarix Orphan, LLC;12 Bow Street;Cambridge, Massachusetts, 02138 February 8, 2016