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AveXis EU Ltd. Granted SME Status by the European Medicines Agency

Dallas – March 19, 2015 – AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced that the European Medicines Agency (EMA) has granted AveXis’ wholly owned European subsidiary, AveXis EU Ltd., SME (Small and Medium Sized Business Entities) status.

The EMA, recognizing the need to encourage drug development by small biotech companies, established the SME program in late 2005 which offers incentives to small and medium business for drug product development. Companies with SME status can receive administrative assistance and reduced/deferred fees associated with regulatory processes, scientific advice, and/or inspections.

“Receiving regulatory guidance directly from the EMA will be invaluable as we move forward with the development of our gene therapy, which is considered an innovative and advanced drug product in the pharmaceutical industry,” commented John A. Carbona, Chief Executive Officer of AveXis.

AveXis has also continued its efforts to grow awareness of the ongoing and upcoming clinical trials with the Contact All Neurologists (C.A.N.) ProgramTM. The C.A.N. ProgramTM has reached neurologists nationwide, informing them of clinical trials available to infants recently diagnosed with SMA Type 1. For neurologists, pediatricians, genetic counselors or physicians interested in participating in the AveXis C.A.N. ProgramTM, please contact

To learn more about the Gene Transfer Clinical Trial, other available trials for SMA, and SMA patient advocacy group, please visit:, a patient-oriented, SMA-focused web resource.

About Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is 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 which codes SMN, a protein necessary for survival of motor neurons. SMA kills more infants than any other genetic disease in today’s world.

About AveXis, Inc.
Based in Dallas, Texas, AveXis is a clinic-stage gene therapy platform company establishing unique industry alliances to create innovative treatments for people with unmet medical needs. Spinal muscular atrophy (SMA) is the company’s first focus.  For more information regarding AveXis, please visit

AveXis Contact:
John A. Carbona, Chief Executive Officer
972-725-7797 or

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