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
Vadastuximab talirine Acute myeloid leukemia Seattle Genetics, Inc.;21823 30th Drive Southeast;Bothell, Washington, 98021 November 10, 2015
Sodium nitrite and EDTA Cystic fibrosis Arch Biopartners, Inc.;27 Saint Clair Avenue East, P. O. Box 305;Toronto, Ontario November 10, 2015
3-{3-[4-(1-aminocyclobutyl)phenyl]-5-phenyl-3H-imidazo[4,5-b]pyridin-2-yl}cpyridin-2-amine bis-mesylate Proteus syndrome ArQule, Inc.;One Wall Street;Burlington, Massachusetts, 01803 November 9, 2015
Human allogeneic bone marrow derived osteoblastic cells Osteogenesis imperfecta Bone Therapeutics SA;37, Rue Auguste Piccard, Gosselies;Gosselies November 9, 2015
Daratumumab Diffuse large B-cell lymphoma Janssen Research and Development, LLC;1400 McKean Road, P. O. Box 776;Spring hOuse, Pennsylvania, 19477 November 9, 2015
Bendamustine oral doseage formulation Chronic lymphocytic leukemia Exinda Theapeutics LLC;8430 Rovana Circle, Suite B;Sacramento, California, 95828 November 5, 2015
Human cystic fibrosis transmembrane conductance regulator (hCFTR) messenger RNA (mRNA) complexed with branched polyethyleneimine (bPEI) Cystic fibrosis Translate Bio;200 Sidney Street, Suite 310;Cambridge, Massachusetts, 02139 November 3, 2015
Cisplatin ChemoThin Wafer Anatomically accessible oral cancers (lip, tongue, gum, floor of mouth, salivary gland, and other oral cavity) Privo Technologies;13 Bristol Street, Unit #1;Cambridge, Massachusetts, 02141 November 3, 2015
Arsenic trioxide capsule (oral) Acute promyelocytic leukemia Orsenix Holdings BV;1105 North Market Street, Suite 1800;Wilmington, Delaware, 19801 November 2, 2015
Recombinant adeno-associated virus serotype 9 vector containing the transgene UBE3A encoding for ubiquitin protein ligase E3A/E6-AP Angelman syndrome Agilis Biotherapeutics, LLC;245 First Street, Suite 1800;Cambridge, Massachusetts, 02142 October 29, 2015
Monoclonal antibody targeting eotaxin-2 Idiopathic pulmonary fibrosis ChemomAb, Ltd.;6 Hanehoset St.;Tel Aviv October 29, 2015
Dabrafenib and trametinib Patients with BRAF mutation positive non-small cell lung cancer Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 October 29, 2015
Lucerastat Fabry disease Idorsia Pharmaceuticals US Inc.;1820 Chapel Avenue West, Suite 150;Cherry Hill, New Jersey, 08002 October 29, 2015
N’-(7-Chloroquinolin-4-yl)-N,N-diethyl-propane-1,3-diamine Malaria Donald J. Krogstad, MD;1430 Tulane Ave., #8317, J.B. Johnson Building;New Orleans, Louisiana, 70112 October 29, 2015
ABCA4 DNA nanoparticles Stargardt Disease Copernicus Therapeutics, Inc.;11000 Cedar Avenue;Cleveland, Ohio, 44106 October 28, 2015
Baclofen Reflex sympathetic dystrophy syndrome Piramal Critical Care, Inc.;261 Brodhead Road, Suite 221;Bethlehem, Pennsylvania, 18017 October 28, 2015
Nano-diamino-tetraiodothyroacetic acid Pancreatic Cancer NanoPharmaceuticals, LLC;1 Discovery Place;Rensselaer, New York, 12144 October 28, 2015
Valsartan oral solution Hypertension in pediatric patients 0 through 16 years of age Carmel Biosciences;5673 Peachtree Dunwoody Road, Suite 440;Atlanta, Georgia, 30342 October 28, 2015
Paricalcitol Pediatric hyperparathyroidism Abbvie, inc.;1 North Waukegan Road;North Chicago, Illinois, 60064 October 27, 2015
Acalabrutinib Waldenstrom macroglobulinemia Acerta Pharma BV;Molenstraat 110, 5342 CC Oss; October 22, 2015
Bucillamine Cystinuria Revive Therapeutics, Ltd.;5 Director Court, Suite 105;Vaughan, Ontario October 22, 2015
Bucillamine Cystinuria Revive Therapeutics, Ltd.;5 Director Court, Suite 105;Vaughan, Ontario October 22, 2015
Deflazacort Pediatric (0 through 16 years of age) juvenile idiopathis arthritis (JIA) International League of Associations for Rheumatology (ILAR) categories excluding systemic JIA PTC Therapeutics, Inc.;100 Corporate Court;South Plainfield, New Jersey, 07080 October 22, 2015
Human soluble receptor-Fc fusion protein that targets human activin A Ovarian cancer Atara Biotherapeutics;4360 Park Terrace Drive, Suite 100;Westlake Village, California, 91361 October 14, 2015
Lisinopril oral solution Hypertension in pediatric patients 0 through 16 years of age Silvergate Pharmaceuticals, Inc.;6251 Greenwood Plaza Blvd., Suite 101;Greenwood Village, Colorado, 80111 October 14, 2015