Fresh from the designation pipeline: orphan drugs recently designated in the European Union (December 2013-February 2014)

Abstract

Introduction: In this issue, designations for hypoparathyroidism, Farber disease, dengue, Rett syndrome, adrenoleukodystrophy, necrotizing enterocolitis, Alagille syndrome, Dravet syndrome, familial intrahepatic cholestasis, familial chylomicronemia, myotonic dystrophy and epidermolysis bullosa are discussed.Areas covered: Orphan drugs designated between December 2013 and February 2014 within the European Union are covered.Expert opinion: Pioglitazone, which was recently designated for treatment of adrenoleukodystrophy, is an example of a drug in which the advantages of the already known pharmacodynamics and preclinical efficacy can be speculated to speed up its development as an orphan drug.