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
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
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
Ublituximab Neuromyelitis optica TG Therapeutics, Inc.;3 Columbus Circle, 15th Floor;New York, New York, 10019 August 25, 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
Phosphoinositide-3-kinase delta inhibitor Chronic lymphocytic leukemia TG Therapeutics, Inc.;3 Columbus Circle, 15th Floor;New York, New York, 10019 August 23, 2016
Etrolizumab Ulcerative colitis Genentech, Inc.;1 Dna Way;South San Francisco, California, 94080 August 22, 2016
Nivolumab Esophageal cancer Bristol-Myers Squibb Co.;5 Research Parkway;Wallingford, Connecticut, 06492 August 22, 2016
Cultured skin substitute (CSS) Burns requiring skin grafting Regenicin Inc.;10 High Court;Little Falls, New Jersey, 07424 August 22, 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
Disufenton sodium Glioblastoma Oblato, Inc.;116 Village Boulevard;Princeton, New Jersey, 08540 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
Live E. coli Nissle bacterium modified to assimilate ammonia Urea cycle disorders Synlogic, Inc.;200 Sidney Street, #320;Cambridge, Massachusetts, 02139 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
Rituximab and recombinant human hyaluronidase Chronic lymphocytic leukemia Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 August 22, 2016