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
Triheptanoin McArdle’s disease Vall d'Hebron Institute of Research July 14, 2016
Sodium hypochlorite Partial deep dermal and full thickness burns Hypo-Stream Ltd July 14, 2016
Volanesorsen sodium Lipodystrophy Ionis USA Limited 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
Dimethyl fumarate Bullous pemphigoid Immungenetics AG July 14, 2016
3-[4-(1H-imidazol-1-ylmethyl)phenyl]-5-(2-methylpropyl)thiophene-2-[(N-butyloxylcarbamate)-sulphonamide] sodium salt Idiopathic pulmonary fibrosis Vicore Pharma AB July 14, 2016
Mifamurtide Alveolar echinococcosis Delta Proteomics SAS July 14, 2016
Adeno-associated viral vector serotype 2.7m8 containing theChrimsonR-tdTomatogene Retinitis pigmentosa GenSight- Biologics July 14, 2016
Mifamurtide Hepatocellular Carcinoma Delta Proteomics SAS 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
Sodium benzoate Ornithine Transcarbamylase Deficiency Lucane Pharma SA 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
Brincidofovir Adenovirus infection in immunocompromised patients Chimerix UK Ltd July 14, 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
[F18] florilglutamic acid Hepatocellular Carcinoma Piramal Imaging S.A.;13 Route de l'Ecole;Matran July 13, 2016
Tolfenamic acid Frontotemporal dementia Nasser H. Zawia;83 Castle Rocks Road;Warwick, Rhode Island, 02886 July 13, 2016
Tolfenamic acid Progressive supranuclear palsy Nasser H. Zawia;83 Castle Rocks Road;Warwick, Rhode Island, 02886 July 13, 2016
Recombinant human biglycan Duchenne muscular dystrophy Tivorsan Pharmaceuticals, Inc.;3 Davol Square, A340;Providence, Rhode Island, 02903 July 13, 2016
[F18] florilglutamic acid Hepatocellular Carcinoma Piramal Imaging S.A.;13 Route de l'Ecole;Matran July 13, 2016
1-(2-chlorophenyl)-1-(S)-hydroxy-2-(S)-carbamoyloxy-propane Status epilepticus Bio-Pharm Solutions Co., Ltd.;Advanced Institute of Convergence Technology, 6F, C Bldg;Yeongtong Suwon July 7, 2016
Brentuximab vedotin Enteropathy-associated T-cell lymphoma Seattle Genetics, Inc.;21823 30th Drive Southeast;Bothell, Washington, 98021 July 7, 2016
Selinexor Soft tissue sarcoma Karyopharm Therapeutics, Inc.;85 Wells Avenue;Newton, Maryland, 02459 July 7, 2016
Deoxythymidine monophosphate and deoxycytidine monophosphate Thymidine kinase 2 deficiency Columbia University Medical Center;Department of Neurology, 630 West 168th Street, P&S; 4-423;New York, New York, 10032 July 7, 2016