AveXis Gains Orphan Drug Designation from the European Medicines Agency for Gene Therapy Treatment in Spinal Muscular Atrophy
AveXis, a leading gene therapy company developing treatments for rare and life-threatening genetic diseases, announced that the European Medicines Agency (EMA) has granted Orphan Drug designation to its gene transfer therapeutic currently in clinical development in the United States for the treatment of type 1 Spinal Muscular Atrophy (SMA). The company received Orphan Drug designation for SMA in the United States in October 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 (EU). There are between 25,000 and 50,000 SMA patients in the United States, 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.
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 (EU). There are between 25,000 and 50,000 SMA patients in the United States, 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.
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