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.