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
Hydrocortisone acetate Ulcerative colitis Cristcot HCA, LLC;555 Virginia Road, Suite 202;Concord, Massachusetts, 01742 June 27, 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
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
Avacopan C3 glomerulopathy ChemoCentryx Limited June 20, 2017
Decitabine and tetrahydrouridine Sickle cell anemia Ulrich Muehlner June 20, 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
Miltefosine Granulomatous amebic encephalitis (GAE) Profounda, Inc.;5790 Hoffner Avenue, Suite 507;Orlando, Florida, 32822 June 19, 2017
NELATIMOTIDE AND ADEGRAMOTIDE combination Myelodysplastic syndrome Boston Biomedical, Inc.;640 Memorial Drive;Cambridge, Massachusetts, 02139 June 19, 2017
Miltefosine Amebiasis Profounda, Inc.;5790 Hoffner Avenue, Suite 507;Orlando, Florida, 32822 June 19, 2017