Fresh from the designation pipeline: orphan drugs recently designated in the European Union (November 2012 – January 2013)

Introduction: Orphan drugs are therapies designated to be used in rare diseases. Orphan drug designation is a regulatory procedure currently available at the European Medicines Agency in the EU and at the Food and Drug Administration (FDA) in the USA. Areas covered: This paper updates and discusses the newly designated orphan drugs in the EU. Orphan drugs for rare disease with no authorised therapy are considered. Expert opinion: Orphan drug development for rare diseases with high unmet therapeutic need (i.e., diseases without approved therapies) should be considered by all players in the pharmaceutical industry. Recently more financial incentives were made available in this area.