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
Recombinant adeno-associated virus vector serotype 9 expressing human N-Sulfoglucosamine Sulfohydrolase Sanfilippo Syndrome Research Institute at Nationwide Children's Hospital;Center for Gene Therapy, 700 Childrens Drive;Columbus, Ohio, 43205 September 1, 2016
Angiotensin (1-7) Recessive dystrophic epidermolysis bullosa (RDEB) Tarix Orphan, LLC;12 Bow Street;Cambridge, Massachusetts, 02138 September 1, 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
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
Humanized IgG4 monoclonal antibody to Toll-Like Receptor 2 (TLR2) Myelodysplastic syndrome Opsona Therapeutics Ltd.;2nd Floor Ashford House, Dublin 2; August 30, 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
Recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues Glycogen storage disease type II (Pompe’s disease) NanoMedSyn August 29, 2016
Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene Duchenne muscular dystrophy Pfizer Limited August 29, 2016
Recombinant human interleukin-12 Acute Radiation Syndrome Acute radiation syndrome Coté Orphan Consulting UK Limited August 29, 2016
Synthetic double-stranded small interfering RNA (siRNA) oligonucleotide targeting delta-aminoevulinic acid synthase 1 Variegate porphyria Alnylam Pharmaceuticals;300 Third Street;Cambridge, Massachusetts, 02142 August 29, 2016
Fimaporfin Bile duct cancer PCI Biotech AS August 29, 2016
Recombinant humanised monoclonal antibody against human complement component C5a Graft Versus Host Disease Alexion Europe SAS August 29, 2016
Masitinib mesilate Amyotrophic lateral sclerosis AB Science August 29, 2016
Sodium benzoate Ornithine translocase deficiency Lucane Pharma SA August 29, 2016
9,10-dimethoxy-5,6-dihydro-[1,3] dioxolo[4,5-g]isoquinolino[3,2-alpha]isoquinolin-7-ium(R)-4-((3R,5S,7S,8R,9S,10S,13R,14S,17R)-3,7-dihydroxy-10,13-dimethyhexadecahydro-1H-cyclopenta[alpha] Primary sclerosing cholangitis HighTide Biopharma Pty. Ltd.;1 Melissa Street;Mount Waverley August 29, 2016
Methotrexate Alkaptonuria aimAKU (Associazione Italiana Malati di Alcaptonuria) August 29, 2016
Venetoclax Multiple myeloma AbbVie Inc.;Regualtory Affairs, 1 North Waukegan Road;North Chicago, Illinois, 60064 August 29, 2016
Nintedanib Systemic scleroderma Boehringer Ingelheim International GmbH August 29, 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
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