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
(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
Ambrisentan/tadalafil fixed combination Pulmonary Arterial Hypertension Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 August 22, 2016
Propofol hemisuccinate Acute repetitive seizures EpaleX Corporation;305 Lytton Avenue;Palo Alto, California, 94301 August 19, 2016
Humanized IgG4 monoclonal antibody that binds to the SEMA4D antigen Huntington disease Vaccinex, Inc.;1895 Mount Hope Avenue;Rochester, New York, 14620 August 16, 2016
Carbon monoxide Idiopathic pulmonary fibrosis Proterris, Inc.;197 West Springfield Street;Boston, Massachusetts, 02118 August 16, 2016
Itraconazole inhalation powder Cystic fibrosis Pulmatrix, Inc;99 Hayden Avenue;Lexington, Massachusetts, 02421 August 16, 2016
Dusquetide Macrophage activation syndrome Soligenix, Inc.;29 Emmons Drive, Suite C-10;Princeton, New Jersey, 08540 August 10, 2016
Retinol palmitate Bronchopulmonary dysplasia Orphanix GmbH;41 Peter-Rosegger-Stra�e;Ried im Innkreis August 8, 2016
Ceramide Hepatocellular Carcinoma Keystone Nano, Inc.;1981 Pine Hall Road;State College, Pennsylvania, 16801 August 8, 2016
Vedolizumab Graft Versus Host Disease Millennium Pharmaceuticals, Inc.;40 Landsdowne Street;Cambridge, Massachusetts, 02139 August 4, 2016
Vemurafenib Erdheim-Chester syndrome Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 August 2, 2016
Melatonin Necrotising enterocolitis Therapicon Srl August 1, 2016
Chimeric antigen receptor directed against the target protein CD4 lentivirally transfected T cells T-cell lymphoma iCell Gene Therapeutics;25 Health Sciences Drive, Suite 118;Stony Brook, New York, 11793 July 28, 2016
Humanized IgG4 monoclonal antibody against total complement component 1, subcomponent s (C1s) Autoimmune hemolytic anemia Bioverativ USA, Inc.;951 Gateway Boulevard;South San Francisco, California, 94080 July 27, 2016
Recombinant, adeno-associated virus serotype 5 vector, containing the transgene, which encodes for the human protein, frataxin Friedreich Ataxia Agilis Biotherapeuties, LLC;245 First Street, Suite 1800;Cambridge, Massachusetts, 02142 July 25, 2016
Flunarizine Hemiplegic migraine Xenon Pharmaceuticals, Inc.;3650 Gilmore Way;Burnaby, British Columbia July 20, 2016
(S)-7-(1acryloylpiperidin-4-yl)-2-(4-phenoxyphenyl)-4,5,6,7-tetrahydropyrazolo[1,5-a]pyrimidine-3-carboxamide Chronic lymphocytic leukemia BeiGene USA, Inc.;2115 Linwood Avenue, 4th Floor;Fort Lee, New Jersey, 07024 July 20, 2016
Pegylated recombinant human interleukin-10 Pancreatic Cancer ARMO BioSciences, Inc.;575 Chesapeake Drive;Redwood City, California, 94063 July 18, 2016
Ruxolitinib Acute lymphoblastic leukemia Incyte Corporation;1801 Augustine Cut-off;Wilmington, Delaware, 19803 July 18, 2016
Adeno-associated viral vector serotype 2.7m8 containing theChrimsonR-tdTomatogene Retinitis pigmentosa GenSight- Biologics July 14, 2016
Autologous Epstein-Barr virus specific T-cells derived from peripheral blood mononuclear cells, expanded ex vivo Extranodal NK/T-cell lymphoma, nasal type Cell Medica Ltd. July 14, 2016
2-[4-(1-methyl-4-pyridin-4-yl-1H-pyrazol-3-yl)-phenoxymethyl]-quinoline succinic acid Huntington disease Pfizer Limited July 14, 2016
Autologous Epstein-Barr virus specific T-cells derived from peripheral blood mononuclear cells, expanded ex vivo Post-transplant lymphoproliferative disorder Cell Medica Ltd. July 14, 2016
Dimethyl fumarate Bullous pemphigoid Immungenetics AG July 14, 2016
Brincidofovir Adenovirus infection in immunocompromised patients Chimerix UK Ltd July 14, 2016