The EMA has granted Orphan Drug designation to a gene transfer therapeutic currently in clinical development in the U.S. for the treatment of type 1 spinal muscular atrophy (SMA). AveXis, a gene therapy company developing treatments for rare and life-threatening genetic diseases, received Orphan Drug designation for SMA in the U.S. in Oct. 2014.
The EMA grants Orphan Drug designation to medicines intended to treat, prevent or diagnose life-threatening and debilitating diseases, with a prevalence no greater than five in 10,000 in the European Union (E.U.). There are between 25,000 and 50,000 SMA patients in the U.S., Europe and Japan combined. Following Orphan Drug designation, sponsors can access a number of incentives including protocol assistance, scientific advice and receive regulatory exclusivity for a ten-year period following approval.