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
Nanoparticulate paclitaxel Ovarian cancer NanOlogy, LLC;231 Bonetti Drive;San Luis Obispo, California, 93401 October 6, 2015
2-amino-2-[2-[2-chloro-4-[[3-(phenylmethoxy) phenyl] thio]phenyl]ethyl]1,3-propanediol hydrochloride Graft Versus Host Disease Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 October 6, 2015
(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 Roivant Sciences GmbH;8 Viaduktstrasse;Basel October 6, 2015
Glyburide Acute spinal cord injury Remedy Pharmaceuticals, Inc.;233 Broadway, Suite 1750;New York, New York, 10279 September 29, 2015
4-Hydroxy-2,2,6,6-tetramethylpiperidine-N-oxyl Cerebral cavernous malformation Recursion Pharmaceuticals, LLC;383 S. Colorow Drive;Salt Lake City, Utah, 84108 September 29, 2015
Aden-associated virus vector serotype 9 expressing human a-L-iduronidase Hurler syndrome REGENXBIO, Inc.;9712 Medical Center Drive, Suite 100;Rockville, Maryland, 20850 September 29, 2015
2-[(4S)-6-(4-chlorophenyl)-1,7,8-trimethylthiophenol[3,2-f]1,2,4-triazolo[4,3-a]1,4-diazepin-4-yl]-N-[3-(4-methylpiperazinyl)propyl]acetamide Nuclear protein in testis (NUT) midline carcinoma Genentech, Inc (a Roche Group Member);1 DNA Way, M/S# 35-5e;South San Francisco, California, 94080 September 29, 2015
Guadecitabine Acute myeloid leukemia Astex Pharmaceuticals, Inc.;4420 Rosewood Drive, Suite 200;Pleasanton, California, 94588 September 29, 2015
Acalabrutinib Mantle cell lymphoma Acerta Pharma BV;Molenstraat 110, 5342 CC Oss; September 21, 2015
Setmelanotide Prader-Willi syndrome Rhythm Metabolics, Inc.;855 Boylston Street, 11th Floor;Boston, Massachusetts, 02116 September 21, 2015
Avelumab Merkel cell cancer EMD Serono Research and Development Institute, Inc.;45A Middlesex Turnpike; September 21, 2015
Adeno-associated viral vector composed of a bioengineered AAV capsid and a codon-optimized expression cassette encoding a high-specific activity variant of human coagulation factor IX Hemophilia B Spark Therapeutics, Inc.;3737 Market Street, Suite 1300;Philadelphia, Pennsylvania, 19104 September 21, 2015
Marizomib Glioma Triphase Research & Development I Corp.;3366 N. Torrey Pines Court, Suite 210;La Jolla, California, 92037 September 21, 2015
Avelumab (Recombinant human monoclonal IgG1 antibody against programmed death ligand-1) Merkel cell cancer EMD Serono Research and Development Institute, Inc.;45A Middlesex Turnpike; September 21, 2015
Recombinant human monoclonal IgG1 antibody against programmed death ligand-1 (anti-PD-L1) Merkel cell cancer EMD Serono, Inc.;45 Middlesex Turnpike;Billerica, Massachusetts, 01821 September 21, 2015
Mecasermin, recombinant human insulin-like growth factor-1 Rett like syndrome Keck Graduate Institute of Applied Life Sciences;535 Watson Drive;Claremont, California, 91711 September 21, 2015
(3S,4R)-3-Ethyl-4-(3H-imidazo[1,2-a]pyrrolo[2,3-e]pyrazin-8-yl)-N-(2,2,2-trifluoroethyl)pyrrolidine-1-carboxamide (2R,3R)-2,3-dihydroxybutanedioate Still’s disease AbbVie, Inc.;1 North Waukegan Road;North Chicago, Illinois, 60064 September 18, 2015
2-Ethylbutyl (2S)-2-{[(S)-{[(2R,3S,4R,5R)-5-(4-aminopyrrolo[2,1-f][1,2,4]triazin-7-yl)-5-cyano-3,4-dihydroxytetrahydrofuran-2-yl]methoxy}(phenoxy)phosphoryl]amino}propanoate Ebola Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 September 18, 2015
Upadacitinib Still’s disease AbbVie, Inc.;1 North Waukegan Road;North Chicago, Illinois, 60064 September 18, 2015
Mononuclear enriched fraction of human umbilical cord blood Amyotrophic lateral sclerosis Saneron CCEL Therapeutics, Inc.;13014 North Dale Mabry Highway, Suite 266;Tampa, Florida, 33618 September 15, 2015
Nivolumab Small cell lung cancer Bristol-Myers Squibb Company;Route 206 & Province Line Road, Mail Stop D32-07;Princeton, New Jersey, 08543 September 15, 2015
Interleukin-1 receptor antagonist anakinra Still’s disease Swedish Orphan Biovitrum AB;SE-112, 75 Stockholm; September 15, 2015
Small molecule FGFR4 inhibitor Hepatocellular Carcinoma Blueprint Medicines Corporation;38 Sidney Street, Suite 200;Cambridge, Massachusetts, 02139 September 14, 2015
Masitinib mesylate Stomach Cancer AB Science;3, avenue George V;Paris September 14, 2015
Immunoglobulin G degrading enzyme of Streptococcus pyogenes Prevention of antibody mediated organ rejection in solid organ transplant patients Hansa Medical AB;P. O. Box 785, SE-220 07 Lund; September 10, 2015