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
(R)-6-(2-fluorophenyl)-N-(3-(2-((2-methoxyethyl)amino)ethyl)phenyl)-5,6-dihydrobenzo[h]quinazolin-2-amine dihydrochloride Bile duct cancer ArQule, Inc.;One Wall Street;Burlington, Massachusetts, 01803 November 19, 2015
Vasomera Duchenne muscular dystrophy PhaseBio Pharmaceuticals, Inc.;1 Great Valley Parkway, Suite 30;Malvern, Pennsylvania, 19355 November 19, 2015
Oxaloacetic acid Amyotrophic lateral sclerosis Terra Biological LLC;3830 Valley Centre Drive, Suite 705-561;San Diego, California, 92130 November 18, 2015
Adeno-associated viral (AAV) vector serotype rh. 10 encoding the human N-sulfoglycosamine sulphohydrolase cDNA (SGSH) Sanfilippo Syndrome LYSOGENE;20 Rue Jacques Dulud;Neuilly-sur-Seine November 18, 2015
4-[(7-Methoxy-2,3-dihydro-1,4-benzothiazepin-4(5H)-yl)methyl]benzoic acid, hemifumarate Duchenne muscular dystrophy ARMGO Pharma, Inc.;777 Old Saw Mill River Road;Tarrytown, New York, 10591 November 18, 2015
Thioureidobutyronitrile Retinoblastoma Innovation Pharmaceuticals, Inc.;100 Cummings Center, Suite 151-B;Beverly, Massachusetts, 01915 November 17, 2015
Adeno-associated virus type 8 delivering a vector genome with human retinoschisin promoter (RS/IRBP) and the human retinoschisin cDNA (hRS) X-linked Juvenile Retinoschisis Paul A. Sievig, MD, PhD, National Eye Institute, NIH;31 Center Drive, Bldg 31, Room 6A03;Bethesda, Maryland, 20892 November 16, 2015
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;N.Zerva 28;Glyfada November 16, 2015
Recombinant adeno-associated virus vector expressing the cyclic nucleotide gated channel alpha subunit (CNGA3) Achromatopsia Applied Genetic Technologies Corp.;11801 Research Drive, Suite D;Alachua, Florida, 32615 November 16, 2015
Diazepam (intranasal) Management of acute repetitive seizures Neurelis Pharmaceuticals, Inc.;1042-B N. El Camino Real, Suite 430;Encinitas, California, 92024 November 16, 2015
N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4 methylpiperazin-1-yl)-2-(tetrahydro-2H-pyran-4-ylamino)benzamide Neuroblastoma Pharma Gateway AB November 11, 2015
Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor Acute lymphoblastic leukaemia Kite Pharma EU B.V. November 11, 2015
Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor Chronic lymphocytic leukaemia/small lymphocytic lymphoma Kite Pharma EU B.V. November 11, 2015
Humanised fusion protein consisting of extracellular domain of CD24 linked to IgG1 Fc domain Graft Versus Host Disease Enpharma Ltd November 11, 2015
Adenovirus associated viral vector serotype 5 containing the humanRPE65gene Leber congenital amaurosis MeiraGTx UK II Limited November 11, 2015
Adenovirus associated viral vector serotype 8 containing the humanCNGB3gene Achromatopsia MeiraGTx UK II Limited November 11, 2015
Sodium phenylbutyrate Pyruvate dehydrogenase complex deficiency Fondazione Telethon November 11, 2015
Humanised monoclonal antibody of the IgG4 kappa isotype targeting CD47 Acute myeloid leukaemia ICON Clinical Research Limited November 11, 2015
Recombinant human interleukin-3 truncated diphtheria toxin fusion protein Blastic plasmacytoid dendritic cell neoplasm TMC Pharma Services Ltd November 11, 2015
Adeno-associated viral vector serotype 8 encoding the humanATP7Bgene under the control of the human alpha-1 antitrypsin promoter Wilson disease Aligen Therapeutics S.L. November 11, 2015
4’-[(2-butyl-4-oxo-1,3-diazaspiro[4.4]non-1-en-3-yl)methyl]-N-(4,5-dimethyl-3-isoxazolyl)-2’-(ethoxymethyl)-[1,1’-biphenyl]-2-sulfonamide Focal segmental glomerulosclerosis Retrophin Europe Limited November 11, 2015
(5S,8S,10aR)-N-benzhydryl-5-((S)-2-(methylamino)propanamido)-3-(3-methylbutanoyl)-6-oxodecahydropyrrolo[1,2-a][1,5]diazocine-8-carboxamide Ovarian cancer ASPHALION, SL November 11, 2015
Interferon alfa-n3 Middle East respiratory syndrome NV Hemispherx BioPharma Europe November 11, 2015
Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor Follicular lymphoma Kite Pharma EU B.V. November 11, 2015
Pentetrazol Idiopathic hypersomnia Balance Therapeutics, Limited November 11, 2015