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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 |
---|---|---|---|---|
Retinol palmitate (vitamin A palmitate) | Retinopathy of prematurity | orphanix GmbH;41 Peter-Rosegger-Stra�e;Ried im Innkreis | June 28, 2017 | |
Chemically modified recombinant sulfamidase | Mucopolysaccharidosis type IIIA (MPS IIIA) | Swedish Orphan Biovitrum AB (publ);D�NSKEBO;Link�ping SV | June 28, 2017 | |
Ganaxolone | Cyclin-dependent kinase-like 5 (CDKL5) gene-related early-onset infantile epileptic encephalopathy | Marinus Pharmaceuticals;170 North Radnor Chester Road, Suite 250;Radnor, Pennsylvania, 19087 | June 28, 2017 | |
Glasdegib | Acute myeloid leukemia | Pfizer, Inc.;445 Eastern Point Road;Groton, Connecticut, 06340 | June 28, 2017 | |
ScAAV9.MeCP2.hCLN3, a self-complementary AAV serotype 9 expressing human CLN3 (hCLN3) | Batten disease | Abeona Therapeutics;6555 Carnegie Blvd, Suite 401;Cleveland, Ohio, 44103 | June 27, 2017 | |
Hydrocortisone acetate | Ulcerative colitis | Cristcot HCA, LLC;555 Virginia Road, Suite 202;Concord, Massachusetts, 01742 | June 27, 2017 | |
N-[(2S)-5-{[(1R,2S)-2-(4-fluorophenyl)cyclopropyl]amino}-1-(4-methylpiperazin-1-yl)-1-oxopentan-2-yl]-4-(1H-1,2,3-triazol-1-yl)benzamide, bis-tosylate salt | Myelofibrosis | Imago BioSciences, Inc.;2729 Debbie Court;San Carlos, California, 94070 | June 26, 2017 | |
Chrysoeriol | Acute myeloid leukemia | Flavocure Biotech;701 East Pratt Street, Suite 2033;Baltimore, Maryland, 21202 | June 22, 2017 | |
Tipifarnib | Multiple myeloma | Paul Maher, MD;10644 Weymouth Street, Suite 104;Bethesda, Maryland, 20814 | June 22, 2017 | |
Humanized anti-BCMA (B-cell maturation antigen) monoclonal antibody | Multiple myeloma | GlaxoSmithKline;980 Great West Road;Brentford-Middlesex | June 22, 2017 | |
[(S)-1-(L-valyl)-N_((S)-1-phenyl-2-(pyridin-2-yl)ethyl)pyrrolidine-2-carboxamide fumarate] | Rett syndrome | Biohaven Pharmaceutics, Inc.;234 Church Street, Suite 304;New Haven, Connecticut, 06510 | June 22, 2017 | |
Two identical 11-mer oligodeoxynucleotide phosphorothioate sequences linked together at their 3′-ends by glycerol-1,3-diphosphorothioate linkages | Melanoma stages IIb-IV | Idera Pharmaceuticals, Inc.;167 Sidney Street;Cambridge, Massachusetts, 02139 | June 21, 2017 | |
Citric acid monohydrate | Acute-on-chronic liver failure | Versantis AG;7 Otto-Stern-Weg, HPL D16.2;Z�rich | June 21, 2017 | |
Ibutamoren mesilate | Growth hormone deficiency | Richardson Associates Regulatory Affairs Ltd | June 20, 2017 | |
Pentamer formyl thiophene acetic acid | Creutzfeldt-Jakob disease | NeuroScios GmbH | June 20, 2017 | |
Recombinant human factor IX protein modified with three point mutations | Hemophilia B | Voisin Consulting S.A.R.L. | June 20, 2017 | |
Sildenafil | Congenital diaphragmatic hernia | Avivia Beheer BV | June 20, 2017 | |
Tripotassium citrate monohydrate and potassium hydrogen carbonate | Distal renal tubular acidosis | Advicenne Pharma SA | June 20, 2017 | |
Synthetic glucagon analogue modified to contain 7 amino acid substitutions | Congenital hyperinsulinism | Zealand Pharma A/S | June 20, 2017 | |
Asp-Arg-Val-Tyr-Ile-His-Pro | Dystrophic epidermolysis bullosa | Envigo Pharma Consulting Ltd | June 20, 2017 | |
Avacopan | C3 glomerulopathy | ChemoCentryx Limited | June 20, 2017 | |
Decitabine and tetrahydrouridine | Sickle cell anemia | Ulrich Muehlner | June 20, 2017 | |
Miltefosine | Amebiasis | Profounda, Inc.;5790 Hoffner Avenue, Suite 507;Orlando, Florida, 32822 | June 19, 2017 | |
Omaveloxolone | Friedreich Ataxia | Reata Pharmaceuticals, Inc.;2801 Gateway Drive, Suite 150;Irving, Texas, 75063 | June 19, 2017 | |
Pamrevlumab | Pancreatic Cancer | FibroGen, Inc.;409 Illinois Street;San Francisco, California, 94158 | June 19, 2017 |