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
Rituximab and recombinant human hyaluronidase Chronic lymphocytic leukemia Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 August 22, 2016
Rituximab and recombinant human hyaluronidase Follicular lymphoma Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 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
Itraconazole inhalation powder Cystic fibrosis Pulmatrix, Inc;99 Hayden Avenue;Lexington, Massachusetts, 02421 August 16, 2016
Carbon monoxide Idiopathic pulmonary fibrosis Proterris, Inc.;197 West Springfield Street;Boston, Massachusetts, 02118 August 16, 2016
Dusquetide Macrophage activation syndrome Soligenix, Inc.;29 Emmons Drive, Suite C-10;Princeton, New Jersey, 08540 August 10, 2016
Ceramide Hepatocellular Carcinoma Keystone Nano, Inc.;1981 Pine Hall Road;State College, Pennsylvania, 16801 August 8, 2016
Retinol palmitate Bronchopulmonary dysplasia Orphanix GmbH;41 Peter-Rosegger-Stra�e;Ried im Innkreis 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
(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
Flunarizine Hemiplegic migraine Xenon Pharmaceuticals, Inc.;3650 Gilmore Way;Burnaby, British Columbia July 20, 2016
Ruxolitinib Acute lymphoblastic leukemia Incyte Corporation;1801 Augustine Cut-off;Wilmington, Delaware, 19803 July 18, 2016
Pegylated recombinant human interleukin-10 Pancreatic Cancer ARMO BioSciences, Inc.;575 Chesapeake Drive;Redwood City, California, 94063 July 18, 2016
Poly(oxy-1,2-ethanediyl), alpha-(carboxymethyl)-omega-methoxy-,amide with arginase 1 [cobalt cofactor] (synthetic human) (1:10), trimer Hyperargininaemia ERA Consulting GmbH July 14, 2016
Recombinant human monoclonal antibody to insulin receptor Congenital hyperinsulinism XOMA UK Limited July 14, 2016
Setmelanotide Pro-opiomelanocortin Deficiency TMC Pharma Services Ltd July 14, 2016
Sirolimus Lymphangioleiomyomatosis Best Regulatory Consulting Ltd July 14, 2016
16-base single-stranded peptide nucleic acid oligonucleotide linked to a 7 aminoacid peptide Soft tissue sarcoma Biogenera SpA July 14, 2016
Three human IgG1 mAbs (REGN3470, REGN3471, and REGN3479) directed against different epitopes on Ebola virus glycoprotein Ebola Regeneron Pharmaceuticals, Inc.;777 Old Saw Mill River Road;Tarrytown, New York, 10591 July 14, 2016