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
Adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and human factor 9 gene (SB-FIX) Hemophilia B Sangamo Therapeutics, Inc.;Point Richmond Tech Center, 501 Canal Boulevard;Richmond, California, 94804 September 1, 2016
Recombinant adeno-associated virus vector serotype 9 expressing human iduronate-2-sulfatase Hunter Syndrome Research Institute at Nationwide Children's Hospital;Center for Gene Therapy, 700 Childrens Drive;Columbus, Ohio, 43205 September 1, 2016
Adeno-associated viral vector differentially expressing microdystrophin 5 gene via the muscle-specific promoter CK8 (rAAV9-CK8-h-�D5) Duchenne muscular dystrophy Solid GT, LLC;161 First Street, Suite 3A;Cambridge, Massachusetts, 02142 August 31, 2016
Amifampridine phosphate Myasthenia gravis Myasthenia gravis Catalyst Pharmaceuticals, Inc.;355 Alhambra Circle, Suite 1250;Coral Gables, Florida, 33134 August 31, 2016
Adeno-associated viral vector differentially expressing microdystrophin 5 gene via the muscle-specific promoter CK8. Duchenne muscular dystrophy Solid GT, LLC;161 First Street, Suite 3A;Cambridge, Massachusetts, 02142 August 31, 2016
Adeno-associated viral vector differentially expressing microdystrophin 5 gene via the muscle-specific promoter CK8. Duchenne muscular dystrophy Solid GT, LLC;161 First Street, Suite 3A;Cambridge, Massachusetts, 02142 August 31, 2016
Humanized IgG4 monoclonal antibody to Toll-Like Receptor 2 (TLR2) Myelodysplastic syndrome Opsona Therapeutics Ltd.;2nd Floor Ashford House, Dublin 2; August 30, 2016
Recombinant protein derived from the saliva of theOrnithodoros moubatatick Paroxysmal nocturnal hemoglobinuria Akari Therapeutics Plc August 29, 2016
Sodium benzoate Lysinuric protein intolerance Lucane Pharma SA August 29, 2016
Valproic Acid McArdle’s disease Vall d'Hebron Institute of Research August 29, 2016
Synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA covalently linked to a ligand containing three N-acetylgalactosamine residues Variegate porphyria Alnylam UK Limited August 29, 2016
S-ethylisothiourea diethylphosphate Symptomatic intradialytic hypotension, including symptomatic hypotension in the immediate post-dialytic period TrioxBio, Inc.;P. O. Box 1000;Wilmington, Delaware, 19801 August 29, 2016
Synthetic ribonucleic acid oligonucleotide directed against superoxide dismutase 1 messenger ribonucleic acid Amyotrophic lateral sclerosis Biogen Idec Limited August 29, 2016
2-((2-ethyl-6-(4-(2-(3-hydroxyazetidin-1-yl)-2-oxoethyl)-piperazin-1-yl)-8-methylimidazo[1,2-alpha]pyridin-3-yl)-(methyl)amino)-4-(4-fluorophenyl)-thiazole-5-carbonitrile Idiopathic pulmonary fibrosis Galapagos NV August 29, 2016
131-I-8H9 monoclonal antibody Neuroblastoma Memorial Sloan Kettering Cancer Center;1275 York Avenue;New York, New York, 10065 August 29, 2016
Temozolomide Glioma Double Bond Pharmaceutical AB August 29, 2016
2-(1,5-dimethyl-3-phenyl-1H-pyrrol-2-yl)-N-{4-[4-(5-fluoro-pyrimidin-2-yl)piperazin-1-yl]-phenyl}-2-oxo-acetamide Scedosporiosis F2G Ltd August 29, 2016
Zoledronic acid Glioma Laboratorio Italiano Biochimico Farmaceutico Lisapharma S.p.A. August 29, 2016
Anti-STAT3 (Signal Transducer and Activator of Transcription 3) B VHH13 single domain antibody Pancreatic Cancer Singh Biotechnology, LLC;4708 Rue Bordeaux;Lutz, Florida, 33558 August 29, 2016
Adenovirus associated viral vector serotype 5 containing the humanRPGRgene Retinitis pigmentosa MeiraGTx UK II Limited August 29, 2016
Autologous mesenchymal stromal cells on a decellularised tracheal scaffold from a cadaveric donor Tracheal stenosis Videregen Ltd August 29, 2016
Cannabidiol Graft Versus Host Disease Richardson Associates Regulatory Affairs Ltd August 29, 2016
Cisplatin Malignant pleural mesothelioma PlumeStars s.r.l. August 29, 2016
L-Pyr-L-Glu-L-Gln-L-Leu-L-Glu-L-Arg-L-Ala-L-Leu-L-Asn-L-Ser-L-Ser Prevention of graft loss in pancreatic islet transplantation Araim Pharma Europe Ltd August 29, 2016
6′-(R)-methyl-5-O-(5-amino-5,6-dideoxy-a-L-talofuranosyl)-paromamine sulfate Hurler syndrome Coté Orphan Consulting UK Limited August 29, 2016