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
Coagulation factor IX (recombinant), Fc fusion protein Hemophilia B Bioverativ Therapeutics, Inc.;225 2nd Avenue;Waltham, Massachusetts, 02451 October 30, 2008
4, 5 dibromorhodamine methyl ester Follicular lymphoma Kiadis Pharma Canada, Inc.;2525 Marie Curie; October 30, 2008
Brentuximab vedotin Anaplastic large cell lymphoma Seattle Genetics, Inc.;21823 30th Drive Southeast;Bothell, Washington, 98021 October 23, 2008
Thawed donor matched banked umbilical cord blood enriched by cell sorting to produce a subpopulation expressing high levels of intracellular aldehyde dehydrogenase To improve patient outcomes by decreasing time to platlet and neutrophil engraftment in patients with inherited metabolic diseases (IMD) undergoing umbilical cord blood transplantation Aldagen, Inc.;2810 Meridian Parkway;Durham, North Carolina, 27713 October 23, 2008
Anti human Nogo-A human monoclonal antibody Acute spinal cord injury Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 October 20, 2008
Epratuzumab Acute lymphoblastic leukemia Immunomedics, Inc.;300 The American Road;Morris Plains, New Jersey, 07950 September 30, 2008
Adeno-associated viral vector serotype 5 containing human ABCA4 gene Stargardt's disease Fondazione Telethon;Via Varese 16 - 00185 Roma; September 30, 2008
Cyclosporine A Prevention of corneal graft rejection Santen SAS;Batiment Genavenir IV, F-91058-Evry cedex; September 30, 2008
Bosentan Idiopathic pulmonary fibrosis Actelion Pharmaceuticals Ltd;Gewerbestrasse 16, CH-4123; September 30, 2008
Canakinumab Pediatric (age 16 and under) juvenile rheumatoid arthritis Novartis Pharmaceuticals Corporation;One Health Plaza, Bldg 135-566G;East Hanover, New Jersey, 07936 September 30, 2008
Difluprednate Endogenous and traumatic anterior uveitis and panuveitis Alcon Pharmaceuticals. Ltd.;Route Des Arsenaux 41, 1701 Fribourg (P.O. Box 61); September 30, 2008
Full Phosphorothioate Antisense Oligonucleotide against EWS-Fli-1 nanoparticles Ewing's sarcoma The Cure Our Children Foundation;1730 Michael Lane;Pacific Palisades, California, 90272 September 22, 2008
Eliglustat Gaucher disease type I Genzyme Corporation;500 Kendall Street;Cambridge, Massachusetts, 02142 September 17, 2008
[131I]-N-(2-(diethylamino) ethyl-4-(4 flurobenzamido)-5-(iodo)-2-methoxybenzamide Metastatic melanoma, stages IIB, IIC, III and IV Molecular Insight Pharmaceuticals, Inc. (Progenics Subsidiary);777 Old Saw Mill River Road;Tarrytown, New York, 10591 September 17, 2008
Human Allogeneic HLA-matched, Umbilical Cord Blood Hematopoietic Progenitor Cells Ex Vivo Expanded on Allogeneic Mesenchymal Precursor Cells Insufficient hematopoietic stem cell production in patients with hematologic malignancies who have failed treatment with conventional chemotherapy Mesoblast, Inc.;275 Madison Avenue, 4th Floor;New York, New York, 10016 September 12, 2008
AQ-13 (4-aminoquinoline analog) Malaria Immtech Pharmaceuticals, Inc.;150 Fairway Drive;Vernon Hills, Illinois, 60061 September 12, 2008
Anti-T-lymphocyte immune globulin, rabbit Prophylaxis of acute allograft rejection in adult recipients in solid organ transplantation Neovii Biotech NA, Inc.;430 Bedford Street, Suite 195;Lexington, Massachusetts, 02420 September 12, 2008
Asfotase alfa Hypophosphatasia Alexion Pharmaceuticals, Inc.;100 College St.;New Haven, Connecticut, 06510 September 12, 2008
Allogeneic mesenchymal precursor cells derived from ex vivo healthy donors harvested from iliac crest. Insufficient hematopoietic stem cell production in patients with hematologic malignancies who have failed treatment with conventional chemotherapy Mesoblast, Inc.;275 Madison Avenue, 4th Floor;New York, NY, 10016 September 12, 2008
Daunorubicin liposomal Acute myeloid leukemia Diatos USA, LLC;15310 Amberly Drive;Tampa, Florida, 33647 September 5, 2008
2-[(3-methyl-4-(2,2,2-trifluoroethoxy) pyridin-2-yl)methylsulfinyl] -1H-benzoimidazole Hepatocellular Carcinoma BioQuant, Inc.;6330 Nancy Ridge Drive;San Diego, California, 92121 August 27, 2008
Cytarabine:daunorubicin liposome injection Acute myeloid leukemia Celator Pharmaceuticals, Inc.;200 Princeton South Corporate Center, Suite 180;Ewing, New Jersey, 08628 August 22, 2008
Seneca Valley virus Neuroendocrine tumors Neotropix, Inc.;351 Phoenixville Pike;Malvern, Pennsylvania, 19355 August 22, 2008
Arenegyr Malignant pleural mesothelioma MolMed S.p.A.;Via Olgettina 58;Milan August 22, 2008
Bardoxolone Pancreatic Cancer Reata Pharmaceuticals, Inc.;2801 Gateway Drive;Irving, Texas, 75063 August 6, 2008