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
Recombinant human interleukin-12 Acute Radiation Syndrome Acute radiation syndrome Coté Orphan Consulting UK Limited August 29, 2016
Recombinant humanised monoclonal antibody against human complement component C5a Graft Versus Host Disease Alexion Europe SAS August 29, 2016
Sodium benzoate Ornithine translocase deficiency Lucane Pharma SA 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
Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene Duchenne muscular dystrophy Pfizer 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
Masitinib mesilate Amyotrophic lateral sclerosis AB Science 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
Valproic Acid McArdle’s disease Vall d'Hebron Institute of Research 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
Recombinant, fully-human, IgG4 immunoglobulin with antigen-binding specificity for human vascular adhesion protein-1 (VAP-1) Primary sclerosing cholangitis Acorda Therapeutics, Inc.;420 Saw Mill River Road;Ardsley, New York, 10502 August 25, 2016
Ublituximab Neuromyelitis optica TG Therapeutics, Inc.;3 Columbus Circle, 15th Floor;New York, New York, 10019 August 25, 2016
Phosphoinositide-3-kinase delta inhibitor Chronic lymphocytic leukemia TG Therapeutics, Inc.;3 Columbus Circle, 15th Floor;New York, New York, 10019 August 23, 2016
(1,3)-�-D-glucan synthesis inhibitor Invasive Aspergillus infections SCYNEXIS, Inc.;101 Hudson Street, Suite 3610;Jersey City, New Jersey, 07302 August 22, 2016
(1,3)-ß-D-glucan synthesis inhibitor Invasive Aspergillus infections SCYNEXIS, Inc.;101 Hudson Street, Suite 3610;Jersey City, New Jersey, 07302 August 22, 2016
Ambrisentan/tadalafil fixed combination Pulmonary Arterial Hypertension Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 August 22, 2016
Stabilized sulforaphane Subarachnoid hemorrhage Evgen Pharma PLC;146 Brownlow Hill;Liverpool August 22, 2016
Disufenton sodium Glioblastoma Oblato, Inc.;116 Village Boulevard;Princeton, New Jersey, 08540 August 22, 2016
Recombinant human interleukin-12 Acute Radiation Syndrome Acute radiation syndrome Neumedicines, Inc.;133 Altadena Drive, Suite 133;Pasadena, California, 91107 August 22, 2016
Live E. coli Nissle bacterium modified to assimilate ammonia Urea cycle disorders Synlogic, Inc.;200 Sidney Street, #320;Cambridge, Massachusetts, 02139 August 22, 2016
Etrolizumab Ulcerative colitis Genentech, Inc.;1 Dna Way;South San Francisco, California, 94080 August 22, 2016
Rituximab and recombinant human hyaluronidase Chronic lymphocytic leukemia Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 August 22, 2016
Rituximab and recombinant human hyaluronidase Follicular lymphoma Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 August 22, 2016