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.

Keywords::

• European Union
• gene therapy
• orphan drug designations
• rare disease