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
Metformin Lafora disease Consorcio Centro de Investigación Biomédica en Red M.P. December 12, 2016
Pegylated recombinant human interleukin-10 Pancreatic Cancer Larode Ltd December 12, 2016
Udenafil Functional single ventricle congenital heart disease Mapi Ireland Limited December 12, 2016
Nintedanib Malignant pleural mesothelioma Boehringer Ingelheim Pharmaceuticals, Inc.;900 Ridgebury Road, P. O. Box 368;Ridgefield, Connecticut, 06877 December 12, 2016
Exendin 9-39 (Competitive antagonist of glucagon-like peptide-1) Hyperinsulinemic hypoglycemia Eiger BioPharmaceuticals, Inc.;350 Cambridge Avenue, Suite 350;Palo Alto, California, 94306 December 8, 2016
Miltefosine Acanthamoeba keratitis Profounda, Inc.;5790 Hoffner Avenue, Suite 507;Orlando, Florida, 32822 December 6, 2016
Miltefosine Primary amebic encephalitis (PAM) Profounda, Inc.;5790 Hoffner Avenue, Suite 507;Orlando, Florida, 32822 December 6, 2016
Propanoic acid, 2,2-dimethyl-,1,1′-[[[[4-[[4-hydroxy-3-(phenylmethyl)phenyl]methyl]-3,5-dimethylphenoxy]methyl]phosphinylidene]bis(oxymethylene)] ester Alport syndrome Viking Therapeutics, Inc.;12340 El Camino Real, Suite 250;San Diego, California, 92130 December 5, 2016
5-{[(3R,4R)-1-(4-chloro-2,6-difluorophenyl)-3,4-dihydroxypiperidin-4-yl]methoxy}-8-fluoro-3,4-dihydroquinolin-2(1H)-one Active tuberculosis Otsuka Pharmaceutical Company, Ltd;2440 Research Blvd;Rockville, Maryland, 20850 December 5, 2016
Cannabidivarin Rett syndrome GW Research Ltd.;Sovereign House, Histon;Cambridge November 30, 2016
Riluzole orally disolving tablet Amyotrophic lateral sclerosis Biohaven Pharmaceutical Holding Company, Ltd;234 Church Street, Suite 304;New Haven, Connecticut, 06511 November 30, 2016
C1 esterase inhibitor (Human) Acute antibody mediated rejection following kidney transplantation Shire ViroPharma, Inc.;300 Shire Way;Lexington, Massachusetts, 02421 November 30, 2016
Yersinia pestis plague recombinant antigen (rF1V; F1 capsular protein fused to V antigen; E. coli) vaccine with aluminum hydroxide Pneumonic Plague DynPort Vaccine Company, LLC, a CSRA Comapny;64 Thomas Johnson Drive;Frederick, Maryland, 21702 November 30, 2016
Lactobacillus plantarum Amyotrophic lateral sclerosis New Biotic, LLC;19546 Rogan Court;Rowland Heights, California, 91748 November 30, 2016
Cannabidiol Graft Versus Host Disease Talent biotechs Ltd.;14-18 Baruch Hirsch Street;Bnei Brak November 29, 2016
Nanoparticles containing Zoledronic acid Glioma Laboratorio Italiano Biochimico Farmaceutico Lisapharma SpA;Via Licinio 11;Erba (CO) November 29, 2016
Cellspan esophageal implant Restore the structure and function of the esophagus subsequent to esophageal damage due to cancer, injury, or congenital abnormality Biostage, Inc.;84 October Hill Road, Suite 11;Holliston, Massachusetts, 01746 November 29, 2016
Risankizumab pediatric Crohn’s disease AbbVie Inc.;Regulatory Affair, 1 North Waukegan Road;North Chicago, Illinois, 60044 November 29, 2016
(2-((2-chlorophenyl)(phenyl)amino)-N-(7-(hydroxyamino)-7-oxoheptyl)pyrimidine-5-carboxamide) Multiple myeloma Acetylon Pharmaceuticals, Inc.;(Celgene Subsidiary), 86 Morris Avenue;Summit, New Jersey, 07901 November 29, 2016
Adenovirus associated viral vector serotype 5 containing the RPGR gene Retinitis pigmentosa MeiraGTx Limited;450 East 29th Street, 15th Floor;New York, New York, 10016 November 29, 2016
Sodium 2-hydroxylinoleate Pancreatic cancer Ability Pharmaceuticals, SL;Edifici Eureka, Campus de la UAB;Bellaterra November 23, 2016
MRNA encoding human ornithine transcarbamylase Ornithine Transcarbamylase Deficiency PhaseRx, Inc.;410 West Harrison Street, Suite 300;Seattle, Washington, 98119 November 23, 2016
5-(4,6-dimorpholino-1,3,5-triazin-2-yl)-4-(trifluoromethyl)-pyridin-2-amine Primary central nervous system lymphoma PIQUR Therapeutics AG;Hochbergerstrasse 60C, CH-4057 Basel; November 21, 2016
Isoindolin-1,3-Di Thione Frontotemporal dementia P2D, Inc.;10101 Alliance Road, Suite 105;Cincinnati, Ohio, 45242 November 21, 2016
Sodium benzoate N-acetylglutamate synthetase deficiency Lucane Pharma SA November 18, 2016