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
Everolimus ointment Tuberous sclerosis Aucta Pharmaceuticals, LLC;675 US Highway One;North Brunswick, New Jersey, 08902 September 10, 2015
Immunoglobulin G degrading enzyme of Streptococcus pyogenes Prevention of antibody mediated organ rejection in solid organ transplant patients Hansa Medical AB;P. O. Box 785, SE-220 07 Lund; September 10, 2015
Mesencephalic, astrocyte-derived neurotrophic factor Retinal artery occlusion Amarantus BioScience Holdings, Inc.;655 Montgomery Street, Suite 900;San Francisco, California, 94111 September 10, 2015
Pentetrazol Idiopathic hypersomnia Balance Therapeutics, Inc.;1250 Bayhill Drive, Suite 125;San Bruno, California, 94066 September 9, 2015
Nimotuzumab Pancreatic cancer InnoCIMab Pte Ltd;21 Bukit Batok Crescent, WCEGA Tower #14-78; September 8, 2015
Hydrocortisone modified release capsules Adrenal insufficiency Diurnal Ltd.;Cardiff Medicentre, Heath Park; September 3, 2015
Nivolumab Hepatocellular Carcinoma Bristol-Myers Squibb Company;P. O. Box 4000;Princeton, New Jersey, 08543 September 2, 2015
Purified autologous type 1 regulatory T lymphocytes specific for human type II collagen Chronic non-infectious uveitis TxCell SA;Allee de la Nertiere, 06560 Valbonne - Sophia-Antipolis; September 1, 2015
Modified a-cobratoxin Pediatric multiple sclerosis Nutra Pharma Corporation;12502 W. Atlantic Blvd;Coral Springs, Florida, 33071 September 1, 2015
Ataluren Aniridia PTC Therapeutics, Inc.;100 Corporate Court;South Plainfield, New Jersey, 07080 September 1, 2015
Udenafil Single ventricle congenital heart disease with Fontan physiology Mezzion Pharma Co. Ltd.;3rd Floor C & H Building, Gangnam-gu 135-879;Seoul August 31, 2015
(S)-N-(5-((R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1-carboxamide hydrogen sulfate Soft tissue sarcoma Loxo Oncology, Inc.;One Landmark Square, Suite 1122;Stamford, Connecticut, 06901 August 31, 2015
Larotrectinib Soft tissue sarcoma Loxo Oncology, Inc.;281 Tresser Boulevard, 9th Floor;Stamford, Connecticut, 06901 August 31, 2015
N-acetylcysteine and sodium thiosulfate Prevention of platinum-induced toxicities in pediatric patients (0 through 16 years of age) Edward A. Neuwelt, MD;Oregon Health & Science University, 3181 SW Sam Jackson Park Road - Mail Code L603;Portland, Oregon, 97239 August 31, 2015
Methotrexate oral solution Oligoarticular juvenile idiopathic arthritis (persistent oligoarthritis, psoriatic juvenile idiopathic arthritis, enthesitis-related arthritis, or undifferentiated arthritis) and polyarticular juvenile idiopathic arthritis in children 0 through 16 years of age Silvergate Pharmaeuticals, Inc.;6251 Greenwood Plaza Blvd, Suite 101;Greenwood Village, Colorado, 80111 August 27, 2015
Nivolumab Glioblastoma Bristol-Myers Squibb Co;P. O. Box 4000;Princeton, New Jersey, 08543 August 27, 2015
Fostamatinib disodium Immune thrombocytopenic purpura Rigel Pharmaceuticals, Inc.;1180 Veterans Boulevard;South San Francisco, California, 94080 August 25, 2015
Adeno-associated virus serotype rh10 vector encoding the human factor IX gene Hemophilia B Dimension Therapeutics;840 Memorial Drive;Cambridge, Massachusetts, 02139 August 25, 2015
Sodium 2-hydroxylinoleate Neuroblastoma Ability Pharmaceuticals, SL;Edifici Eureka, Campus de la UAB;Bellaterra August 25, 2015
Humanized monoclonal antibody of the IgG4 kappa isotype targeting CD47 Acute myeloid (myelogenous) leukemia Forty Seven, Inc.;1490 O'Brien Drive, Suit A;Menlo Park, California, 94025 August 20, 2015
Daratumumab Mantle cell lymphoma Janssen Research & Development, LLC;1400 McKean Road, P. O. Box 776;Spring House, Pennsylvania, 19477 August 20, 2015
Encapsulated spores from fecal microbiota Recurrent Clostridium difficile infection (CDI) Seres Therapeutics, Inc.;200 Sidney Street;Cambridge, Massachusetts, 02139 August 19, 2015
Human allogeneic bone-marrow-derived osteoblastic cells Osteogenesis imperfecta Bone Therapeutics SA August 10, 2015
Glycyl-L-2-methylprolyl-L-glutamic acid Rett syndrome QRC Consultants Ltd August 10, 2015
Recombinant human acid ceramidase Cystic fibrosis Enzyvant Farber Ireland Limited August 10, 2015