Treatments

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

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Country Product Indication Sponsor Date
Brincidofovir Adenovirus infection in immunocompromised patients Chimerix UK Ltd July 14, 2016
Mifamurtide Alveolar echinococcosis Delta Proteomics SAS 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
Mifamurtide Hepatocellular Carcinoma Delta Proteomics SAS July 14, 2016
Recombinant human monoclonal antibody to insulin receptor Congenital hyperinsulinism XOMA UK Limited July 14, 2016
Sodium benzoate Ornithine Transcarbamylase Deficiency Lucane Pharma SA 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
Sodium benzoate Carbamoyl phosphate synthase 1 deficiency Lucane Pharma SA July 14, 2016
Autologous CD4+ and CD8+ T-cells transduced with lentiviral vector containing an affinity-enhanced T-cell receptor targeting the New York esophageal antigen-1 Soft tissue sarcoma Adaptimmune Limited July 14, 2016
Sodium benzoate Citrullinaemia type 1 Lucane Pharma SA July 14, 2016
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
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
[F18] florilglutamic acid Hepatocellular Carcinoma Piramal Imaging S.A.;13 Route de l'Ecole;Matran 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
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
Brentuximab vedotin Enteropathy-associated T-cell lymphoma Seattle Genetics, Inc.;21823 30th Drive Southeast;Bothell, Washington, 98021 July 7, 2016