AveXis Receives Orphan Drug Status from the US Food and Drug Administration (FDA) for chariSMA for the Treatment of Spinal Muscular Atrophy

Dallas, TX, October 04, 2014 --(PR.com)-- AveXis Inc., a synthetic biology platform company primarily focused on developing treatments for Spinal Muscular Atrophy (SMA) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its product scAAV9, called chariSMA to treat Spinal Muscular Atrophy patients.

SMA is an autosomal recessive birth defect, and the largest genetic killer of infants. It is also a disease that has no know treatment or cure. AveXis is currently engaged in an FDA sanctioned Phase 1 clinical trial at Nationwide Children’s Hospital in Columbus, Ohio (clinicaltrials.gov Identifier: NCT02122952).

Orphan Drug Designation is granted by the FDA Office of Orphan Drug Products to drugs intended to treat a rare disease or condition affecting fewer than 200,000 people in the U.S. There are between 25,000 and 50,000 SMA patients in the U.S., Europe and Japan. Typically type 1 patients do not live beyond 2 years of age. No cure or treatment is available for this disease beyond basic respiratory and palliative care.

ChariSMA, the gene therapy drug product AveXis is developing delivered intravenously is a genetically-modified virus that inserts a replacement gene called the Survival Motor Neuron gene (SMN), using licensed technology from ReGenX Biosciences and the Research Institute at Nationwide Children’s Hospital and others.

“SMA is a devastating disease for everyone it touches. Our technology is very simple to describe. These unfortunate infants are born with a mutated or malfunctioning gene on the 5th chromosome, reducing production of a key protein called SMN which stands for survival motor neuron. SMN is required in motor neurons that control muscles that are involved in breathing, sitting up and swallowing, without it these children pass away, it is my hope that the incredible pre-clinical work pioneered by Dr. Brian Kaspar and others will translate into humans,” said John A. Carbona, Chief Executive Officer of AveXis.

AveXis is also working with leading researches in European centers to understand the financial and regulatory pathways forward to initiate a mirror image trial in Europe in 2015.

The Orphan Drug designation is granted to development-stage novel therapeutics that offer potential value in the treatment of rare diseases and medical conditions that affect fewer than 200,000 patients in the US. Orphan Drug designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received Orphan Drug designation is not subject to the Prescription Drug User Fee Act (PDUFA) fees unless the application includes an indication for other than the rare disease or condition for which the therapeutic was designated. In addition AveXis could qualify for a Rare Pediatric Disease Priority Review Voucher (PRV).

The priority review voucher is an expedited review granted by the Food and Drug Administration (FDA) to the developer of a treatment for neglected diseases.

About Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal-recessive genetic disorder characterized by progressive weakness of the lower motor neurons. 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-ready synthetic biology 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 www.avexisinc.com

About Nationwide Children’s Hospital
Ranked 7th of only 10 children's hospitals on U.S. News & World Report’s 2014-15 “America’s Best Children’s Hospitals Honor Roll,” Nationwide Children’s Hospital is one of the nation’s largest not-for-profit freestanding pediatric healthcare networks providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease. Nationwide Children’s, also ranked among the Top 10 on Parents magazine’s 2013 “Best Children’s Hospitals” list, has a medical staff of 1,100 and nearly 10,000 employees providing state-of-the-art pediatric care for one million patient visits annually. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s physicians train the next generation of pediatricians and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded freestanding pediatric research facilities. Nationwide Children’s remains true to the original mission since its founding in 1892 of providing care regardless of a family’s ability to pay. More information is available at NationwideChildrens.org.
AveXis Inc.
Jillian Bowman