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AveXis Gains Orphan Drug Designation from the European Medicines Agency for Gene Therapy Treatment in Spinal Muscular Atrophy

Dallas – June 23, 2015 – AveXis, Inc., a leading gene therapy company developing treatments for rare and life-threatening genetic diseases, today 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.

“Receiving Orphan Drug designation for our investigational medicine in the EU is an important regulatory milestone in the advancement of our development program for SMA,” said Sean P. Nolan, chief executive officer of AveXis. “We look forward to continuing dialogue with global regulators as we work to develop this treatment for patients suffering from SMA around the world.”

About AveXis, Inc.
AveXis is a clinical-stage gene therapy company establishing unique industry alliances to create innovative treatments in areas of unmet medical needs. The company’s most advanced product candidate, ChariSMA, is in a fully-enrolled Phase 1 clinical trial for the treatment of spinal muscular atrophy (SMA), an autosomal-recessive genetic disorder characterized by lower motor neuron loss and progressive muscle weakness. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. SMA is the most common cause of infant mortality and is the second-most common autosomal recessive genetic disorder. For more information about AveXis, please visit

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Lauren Barbiero
W2O Group