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
Metavert, N-hydroxy-4-(5-((4-methoxyphenyl)amino)-1,3,4-oxadiazol-2-yl)benzamide Pancreatic Cancer Avenzoar Pharmaceuticals, Inc.;662 Encinitas Blvd, Suite 248;Encinitas, California, 92024 July 6, 2016
Anti-HER2 bispecific antibody drug conjugate Ovarian cancer Zymeworks Inc.;18 West Mercer Street, Suite 370;Seattle, Washington, 98119 July 6, 2016
Nintedanib Systemic scleroderma Boehringer Ingelheim Pharmaceuticals, Inc.;900 Ridgebury Road, Box 368;Ridgefield, Connecticut, 06877 July 6, 2016
Mazindol Narcolepsy NLS-0 Pharma AG;10 Breitenweg, CH-6370;Stans NW July 6, 2016
Modified mRNA encoding UGT1A1 protein, formulated in lipid nanoparticles Crigler-Najjar syndrome Moderna Therapeutics, Inc.;320 Bent Street;Cambridge, Massachusetts, 02141 July 5, 2016
Bortezomib Neurofibromatosis type 2 BioXcel Corporation;780 East Main Street, Suite 2;Branford, Connecticut, 06405 July 5, 2016
Adenovirus-associated viral vector serotype 5 containing the human pde6B gene Retinitis pigmentosa HORAMA, SAS;9 Rue de l'�peron;Paris July 5, 2016
Small molecule Ewing Sarcoma breakpoint region 1/Friend leukemia virus integration 1 fusion protein inhibitor Ewing sarcoma Tokalas, Inc.;P. O. Box 1269;Rancho Santa Fe, California, 92067 June 30, 2016
Autologous osteogenic cells cultured from adipose tissue derived stem cells embedded in extracellular matrix with demineralized bone matrix. Congenital pseudarthrosis of the tibia Novadip Biosciences;11 Granbonpr�;Mont-Saint-Guibert June 30, 2016
Peptide consisting of L-amino acids with the sequence H-lys-glu-phe-leu-his-pro-ser-lys-val-asp-leu-pro-arg-OH Pancreatic cancer Soricimed Biopharma, Inc.;18 Botsford Street, Suite 201;Moncton, New Brunswick June 29, 2016
Recombinant, glycoenfineered humanized IgG1 kappa monoclonal antibody directed against Fibroblast Growth Factor Receptor 2b Stomach Cancer Five Prime Therapeutics, Inc.;2 Corporate Drive;South San Francisco, California, 94080 June 29, 2016
(S)-7-(1-acryloylpiperidin-4-yl)-2-(4-phenoxyphenyl)-4,5,6,7-tetrahydropyrazolo[1,5-a]pyrimidine-3-carboxamide Waldenstrom macroglobulinemia BeiGene USA, Inc.;2115 Linwood Avenue, 4th Floor;Fort Lee, New Jersey, 07024 June 29, 2016
Humanised monoclonal antibody targeting interleukin-15 Eosinophilic esophagitis Eosinophilic esophagitis Calypso Biotech B.V. June 27, 2016
Setmelanotide Prader-Willi syndrome TMC Pharma Services Ltd June 27, 2016
Molgramostim Acute respiratory distress syndrome Savara ApS June 27, 2016
Recombinant humanised monoclonal IgG2 lambda antibody against human sclerostin Osteogenesis imperfecta Mereo Biopharma 3 Limited June 27, 2016
Teriparatide Hypoparathyroidism Alacrita LLP June 27, 2016
Triclabendazole Fascioliasis Tropicalis Pharmaceuticals, LLC;22345 Bracketts Road;Excelsior, Minnesota, 55331 June 27, 2016
High molecular weight multimer of E3-Fc, an endostatin C-terminus and IgG1-Fc fusion protein Systemic scleroderma iBio, Inc.;600 Madison Avenue, Suite 1601;New York, New York, 10022 June 27, 2016
Melatonin Neonatal sepsis Therapicon Srl June 27, 2016
Modified mRNA encoding the UGT1A1 protein Crigler-Najjar syndrome Alexion Europe SAS June 27, 2016
Recombinant protein derived from the saliva of theOrnithodoros moubatatick Guillain-Barré syndrome Akari Therapeutics Plc June 27, 2016
3-(5-amino-2-methyl-4-oxoquinazolin-3(4H)-yl)piperidine-2,6-dione hydrochloride Diffuse Large B-cell Lymphoma Celgene Europe Limited June 27, 2016
Allogeneic donor-derived ex-vivo expanded T lymphocytes transduced with a retroviral vector containing inducible caspase 9 and truncated CD19 Treatment in haematopoietic stem cell transplantation Bellicum Pharma Limited June 27, 2016
Citric acid monohydrate Acute liver failure CATS Consultants GmbH June 27, 2016