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
(6aR, 10aR)-3-(1⿿,1⿿-dimethylheptyl)-ο8-tetrahydro-cannabinol-9-carboxylic acid Systemic scleroderma Corbus Pharmaceuticals, Inc.;100 River Ridge Drive, Suite 103;Norwood, Massachusetts, 02062 June 10, 2015
(6aR, 10aR)-3-(1⿿,1⿿-dimethylheptyl)-ο8-tetrahydro-cannabinol-9-carboxylic acid Systemic scleroderma Corbus Pharmaceuticals, Inc.;100 River Ridge Drive, Suite 103;Norwood, Massachusetts, 02062 June 10, 2015
Staphylococcal aureus protein A Immune thrombocytopenic purpura Protalex, Inc.;131 Columbia Turnpike, Suite 1;Florham Park, New Jersey, 07932 June 10, 2015
Recombinant human CD24FcIg (humanized fusion protein consisting of the extracellular domain of CD24 linked to IgG1 Fc domain Graft Versus Host Disease OncoImmune, Inc.;502 E Street, NE;Washington, District of Columbia, 20002 June 9, 2015
Fully human IgG2 monoclonal antibody that binds insulin receptors Congenital hyperinsulinism XOMA (US) LLC;2910 Seventh St.;Berkeley, California, 94710 June 9, 2015
Tumor-inflitrating lymphocytes; LN-144 Malignant melanoma Stages IIb to IV Iovance Biotherapeutics, Inc.;999 Skyway Road, Suite 150;San Carlos, California, 94070 June 9, 2015
Ivosidenib Acute myeloid leukemia Agios Pharmaceuticals, Inc.;88 Sidney Street;Cambridge, Massachusetts, 02139 June 9, 2015
Isocitrate dehydrogenase 1 (IDH1)-mutant inhibitor Acute myeloid leukemia Agios Pharmaceuticals, Inc.;88 Sidney Street;Cambridge, Massachusetts, 02139 June 9, 2015
Double stranded oligomer AD00370 RNA interference-based liver targeted therapeutic Alpha-1 antitrypsin deficiency Arrowhead Research Corporation;225 South Lake Avenue, Suite 1050;Pasadena, California, 91101 June 9, 2015
Adenoviral vector expressing the E.coli purine nucleoside phosphorylase gene and fludarabine Intratumoral treatment of anatomically accessible oral and pharyngeal cancers (lip, tongue, gum, floor of mouth, salivary gland, and other oral cavity) PNP Therapeutics, Inc.;15 Richard Arrington Jr. Blvd North;Birmingham, Alabama, 35203 June 8, 2015
Regorafenib Hepatocellular Carcinoma Bayer HealthCare Pharmaceuticals, Inc.;100 Bayer Blvd, P. O. Box 915;Whippany, New Jersey, 07981 June 4, 2015
Ibudilast Krabbe disease MediciNova, Inc.;4275 Executive Square, Suite 650;La Jolla, California, 92037 June 1, 2015
Adult hemogenic endothelial cells Aplastic anemia aplastic anemia HemoGenyx LLC;640 Elliott Street, Suite 108;Buffalo, New York, 14203 May 27, 2015
Prasugrel hydrochloride Sickle cell anemia Eli Lilly;Lilly Corporate Center;Indianapolis, Indiana, 46285 May 26, 2015
Lutetium (177Lu)-edotreotide Gastro-entero-pancreatic neuroendocrine tumors ITM Solucin GmbH;Lichtenbergstrasse 1, 85748 Garching; May 21, 2015
Fusion proteins composed by a genetically modified cholera toxin subunit A1, peptides from the acetylcholine receptor alpha chain and a dimer of the D fragment fromStaphylococcus aureusprotein A Myasthenia gravis Myasthenia gravis Toleranzia AB May 21, 2015
Reduced oxydised N-acetyl heparin Multiple myeloma Leadiant Biosciences Ltd May 21, 2015
AASSGVSTPGSAGHDIITEQPRS Huntington disease Centre National de la Recherche Scientifique (CNRS) May 21, 2015
Trehalose Oculopharyngeal muscular dystrophy Dr Ulrich Granzer May 21, 2015
5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride Huntington disease Prana Biotechnology UK Limited May 21, 2015
Humanised anti-CD37 monoclonal antibody conjugated to maytansinoid DM1 Diffuse Large B-cell Lymphoma Voisin Consulting S.A.R.L. May 21, 2015
2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol-6-yl)acetamide Cystic fibrosis Clinical Network Services (UK) Ltd May 21, 2015
Echinomycin Acute myeloid leukemia OncoImmune, Inc.;502 E. Street, NE;Washington, District of Columbia, 20002 May 21, 2015
Triheptanoin Glucose transporter type-1 deficiency syndrome Ultragenyx UK Limited May 21, 2015
Adult human bone-marrow-derived, ex-vivo-expanded, pooled allogeneic mesenchymal stromal cells Buerger’s disease Regulatory Resources Group Ltd May 21, 2015