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
Arimoclomol Inclusion Body Myositis Orphazyme ApS;Ole Maaloes Vej 3, 2200 Copenhagen; November 2, 2017
Replication defective herpes simplex type 1 virus (HSV-1) vector expressing human COL7 protein Dystrophic epidermolysis bullosa Krystal Biotech, LLC;2100 Wharton Street, Suite 701;Pittsburgh, Pennsylvania, 15203 November 2, 2017
Baricitinib Pediatric systemic lupus erythematosus Eli Lilly and Company;Lilly Corporate Center;Indianapolis, Indiana, 46285 November 2, 2017
Live bacterial product consisting of defined consortia of Clostridium clusters live bacterial product consisting of defined consortia of Clostridium clusters Prevention of recurrent Clostridium difficile infection in individuals with prior recurrent Clostridium difficile infection Vedanta Biosciences, Inc.;19 Blackstone Street, Third Floor;Cambridge, Massachusetts, 02139 November 2, 2017
1-(fluoro-18F)-3-((2-((1E,3E)-4-(6-(methylamino)pyridin-3-yl)buta-1,3-dien-1-yl)benzo[d]thiazol-6-yl)oxy)propan-2-ol Progressive supranuclear palsy Aprinoia Therapeutics;17F, No. 3 Yuanqu Street;Nangang District November 1, 2017
Vigabatrin and cosyntropin West syndrome West Therapeutic Development, LLC;540 Ziegler Drive;Grayslake, Illinois, 60030 November 1, 2017
(+)-1-(3-(aminomethyl) phenyl)-N-(5-((3-cyanophenyl)(cyclopropylmethylamino)methyl)-2-fluorophenyl)-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide dihydrochloride C1-inhibitor-dependent angioedema (including prevention and treatment of attacks) BioCryst Pharmaceuticals, Inc.;4505 Emperor Blvd, Suite 200200;Durham, North Carolina, 27703 November 1, 2017
Indoximod Metastatic Melanoma NewLink Genetics Corporation;2503 South Loop Drive, Suite 5100;Ames, Iowa, 50010 October 26, 2017
Copper sulfate Keratoconus iVeena Drug Delivery Systems, Inc.;391 Chipeta Way, Suite I;Salt Lake City, Utah, 84108 October 26, 2017
Seribantumab Non-small cell lung cancer Merrimack Pharmaceuticals, Inc.;1 Kendall Square, Suite B7201;Cambridge, Massachusetts, 02139 October 25, 2017
9-cis,12-cis-11,11-D2-linoleic acid ethyl ester PLA2G6-associated neurodegeneration Retrotope, Inc.;4300 El Camino Real, Suite 201;Los Altos, California, 94022 October 24, 2017
Glasdegib Myelodysplastic syndrome Pfizer, Inc.;445 Eastern Point Road;Groton, Connecticut, 06340 October 20, 2017
Glepaglutide Short bowel syndrome Zealand Pharma A/S;36 Smedeland;Glostrup October 20, 2017
Valbenazine Tourette syndrome Neurocrine Biosciences, Inc.;12780 El Camino Real;San Diego, California, 92130 October 19, 2017
A strain of modified live probiotic bacterium Escherichia coli (E.coli) Nissle 1917 that has been modified to treat phenylketonuria by consuming phenylalanine within the gastrointestinal tract Phenylketonuria Synlogic, Inc.;200 Sidney Street, Suite 320;Cambridge, Massachusetts, 02139 October 18, 2017
Vatiquinone Pontocerebellar Hypoplasia BioElectron Technology Corporation;350 North Bernardo Avenue;Mountain View, California, 94043 October 18, 2017
Glucopyranosyl lipid A Follicular lymphoma Immune Design Ltd October 16, 2017
Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F Multiple myeloma GlaxoSmithKline Trading Services Limited October 16, 2017
Ofranergene obadenovec Ovarian cancer Envigo Pharma Consulting Ltd October 16, 2017
Recombinant adeno-associated viral vector serotype 5 encodingStaphylococcus aureusCas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of theCEP290gene Leber congenital amaurosis Pharma Gateway AB October 16, 2017
Autologous human peripheral blood mononuclear cells incubated ex vivo with protein TBX-4000 (Tat-MYC) Malignant melanoma Stages IIb to IV Taiga Biotechnologies, Inc.;12635 Montview Boulevard, Fitzsimmons BBioscience Park Center;Aurora, Colorado, 80045 October 16, 2017
Entospletinib Acute myeloid leukaemia Gilead Sciences International Ltd. October 16, 2017
Seladelpar Primary biliary cirrhosis Larode Ltd October 16, 2017
Glasdegib maleate Acute myeloid leukaemia Pfizer Limited October 16, 2017
Synthetic cyclic 8 amino acid analogue of human unacylated ghrelin Prader-Willi syndrome Alizé Pharma October 16, 2017