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

Abstract

Introduction: In this issue, designations for myotonic disorders, sarcoidosis, complex regional pain syndrome, Allan-Herndon-Dudley disease, Duchenne muscular dystrophy, Wiskott-Aldrich syndrome, eosinophilic esophagitis, spinal cord injury and Stargardt syndrome are discussed. These are all rare diseases with a highly unmet therapeutic need.

Areas covered: This article will outline the orphan drugs designated between October and November 2013 within the European Union.

Expert opinion: Various compounds or biological therapies were designated for rare diseases and some of them are compounds traditionally used for other non-rare indications. If they are going to become authorised on the market as orphan drugs, then they can represent cheaper therapeutic alternatives able to reach most of the target population.

Keywords::

• European Union
• expert opinion
• orphan drug designations
• rare disease