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
Fast skeletal muscle troponin activator Spinal muscular atrophy Cytokinetics, Inc.;280 East Grand Avenue;South San Francisco, California, 94080 May 11, 2017
2-amino-1-[2-(4-fluoro-phenyl)-3-(4-fluoro-phenyl)amino)-8,8-dimethyl-5,6-dihydro-imidazo[1,2-a]pyrazin-7(8H)-yl)-ethanone diphosphate Malaria Novartis Pharmaceuticals Corporation;1 Health Plaza;East Hanover, New Jersey, 07936 May 11, 2017
Povidone-iodine 0.5% (w/w) in dimethylsulfoxide 44% (w/w) Microbial keratitis (which includes Acanthamoeba keratitis) Veloce BioPharma LLC;7008 Lafayette Avenue;Fort Washington, Pennsylvania, 19034 May 11, 2017
Ritanserin Glioblastoma The University of Virginia/Benjamin Purow;1215 Lee Street;Charlottesville, Virginia, 22908 May 11, 2017
Rilonacept Deficiency of interleukin-1 receptor antagonist (DIRA) Regeneron Pharmaceuticals, Inc.;777 Old Saw Mill River Road;Tarrytown, New York, 10591 May 9, 2017
Humanized anti-CD19, Fc engineered, monoclonal antibody with enhanced binding to FcyRIIb Immunoglobulin G4-related disease Xencor, Inc.;111 West Lemon Avenue;Monrovia, California, 91016 May 9, 2017
Larotrectinib Solid tumors with NTRK-fusion proteins Loxo Oncology, Inc.;281 Tresser Boulevard, 9th Floor;Stamford, Connecticut, 06901 May 9, 2017
Autologous CD34+ hematopoietic stem cells transduced with an hCD11b lentiviral vector encoding for the human N-sulfoglucosamine sulfohydrolase (SGSH) gene Mucopolysaccharidosis IIIA Orchard Therapeutics North America;1118 Chess Drive, Suite A;Foster City, California, 94404 May 3, 2017
Recombinant adeno-associated viral vector, AAV2 serotype 6 (rAAv2/6), expressing human factor 8 (hf8) cDNA Hemophilia A Sangamo Therapeutics, Inc.;Point Richmond Tech Center, 501 Canal Boulevard;Richmond, California, 94804 May 3, 2017
Cisplatin loaded hyaluronate film for implant Malignant pleural mesothelioma PlumeStars s.r.l.;Via Lago Scuro11, 43124 Parma; May 2, 2017
Macromolecular conjugate of heparin sodium on a polymer backbone Prevention of ischemia-reperfusion injury associated with solid organ tranplantation Corline Biomedical AB;5 Lefflersgatan;Uppsala May 2, 2017
Estetrol Neonatal encephalopathy Mithra Pharmaceuticals S.A. April 28, 2017
Mutated isocitrate dehydrogenase 1 (IDH1) inhibitor Acute myeloid leukemia FORMA Therapeutics, Inc.;500 Arsenal Street, Suite 100;Watertown, Massachusetts, 02472 April 27, 2017
Ivosidenib Bile duct cancer Agios Pharmaceuticals, Inc.;88 Sidney Street;Cambridge, Massachusetts, 02139 April 26, 2017
Ivosidenib Bile duct cancer Agios Pharmaceuticals, Inc.;88 Sidney Street;Cambridge, Massachusetts, 02139 April 26, 2017
Small molecule Mas receptor agonist Duchenne muscular dystrophy RASRx, LLC;231 Bonetti Drive, Suite 240;San Luis Obispo, California, 93401 April 25, 2017
(S)-8-{2-amino-6-[1-(5-chloro-biphenyl-2-yl)-(R)-2,2,2-trifluoro-ethoxy]-pyrimidin-4-yl}-2,8-diaza-spiro[4.5]decane-3-carboxylic acid ethyl ester Pulmonary Arterial Hypertension Biological Consulting Europe Ltd April 20, 2017
Autologous adult bone marrow-derived non-expanded CD133+ haematopoietic stem cells Asherman's syndrome Igenomix, S.L. April 20, 2017
Autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation antigen with 4-1BB and CD3-zeta intracellular signalling domains Multiple myeloma Celgene Europe Limited April 20, 2017
Emeramide Prevention of mercury toxicity NBMI Science Limited April 20, 2017
Human normal immunoglobulin Treatment in solid organ transplantation Hôpital Foch April 20, 2017
Modified messenger ribonucleic acid encoding human ornithine transcarbamylase enzyme encapsulated into lipid nanoparticles Ornithine Transcarbamylase Deficiency PhaseRx Ireland, Ltd April 20, 2017
N-[(1R)-1-phenylethyl]-6-{1H-pyrazolo[3,4-d]pyrimidin-4-yl}quinazolin-2-amine Fragile X syndrome Sentinel Oncology Limited April 20, 2017
Rituximab Treatment in solid organ transplantation Hôpital Foch April 20, 2017
Thymidine and deoxycytidine Mitochondrial DNA depletion syndrome, myopathic form Vall d'Hebron Institute of Research April 20, 2017