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.

Become a Partner.

Country Product Indication Sponsor Date
(2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridin-2-yl)-4-fluoropyrrolidine-2-carboxamide Hemoglobinuria Achillion Pharmaceuticals, Inc;300 George Street;New Haven, Connecticut, 06511 November 2, 2017
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
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
Adenoviral vector of serotype 5 modified to contain a chimeric sequence consisting of a minimal urokinase-type plasminogen activator receptor promoter preceded by three Notch-responsive elements, and coated with oligopeptide end-modified poly (beta-amino) esters Pancreatic Cancer Sagetis Biotech, S.L. October 16, 2017
Autologous ex-vivo-expanded peripheral polyclonal lymphocytes enriched in activated natural killer cells Multiple myeloma CellProtect Nordic Pharmaceuticals AB October 16, 2017
Cannabidiol West syndrome GW Research Ltd October 16, 2017
Upamostat Pancreatic Cancer RedHill Biopharma Ltd.;21 Ha'arba'a Street;Tel Aviv-Yafo October 16, 2017
Bitopertin Beta-thalassaemia intermedia and major Roche Registration Limited October 16, 2017
Cannabidivarin Rett syndrome GW Research Ltd October 16, 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