AveXis Files for FDA Approval of Gene Therapy to treat Spinal Muscular Atrophy Type I

CHICAGO - Oct. 18, 2018 - PRLog -- AveXis, Inc., a Novartis company, today announced that they have filed for FDA approval of AVXS-101, a gene therapy that replaces the survival motor neuron 1 (SMN1) gene, which is missing or mutated in individuals with SMA.

This first filing is for intravenous (IV) delivery of gene therapy, a method of delivery that can be used to treat infants affected by spinal muscular atrophy type I.

Spinal muscular atrophy (SMA) is a neuromuscular disease caused by a mutation in the gene survival motor neuron 1. In a healthy person, this gene produces a protein that is critical to the function of the nerves that control muscles. Without it, those nerve cells cannot properly function and eventually die, leading to debilitating and often fatal muscle weakness.

SMA type I is the most severe and most common form, accounting for approximately 60% of all cases of SMA. If not treated, SMA type I is typically fatal within the first two years of life. It is currently the leading genetic cause of death for infants under two years of age.

In addition, AveXis is currently testing intrathecal (IT) delivery of AVXS-101 in clinical trials. IT delivery would allow older patients with SMA type II, III or IV to receive the therapy. While these forms of the disease are typically less severe than SMA type I, they impact the individual's ability to walk, eat and breathe on his or her own.

"We congratulate AveXis and Novartis on reaching this milestone," said Kenneth Hobby, president of Cure SMA, the leading national non-profit dedicated to the treatment and cure of spinal muscular atrophy. "The IV clinical trial data shows that a one-time dose of gene therapy has the potential for a transformative impact on this life-threatening disease. We look forward to a rapid approval by the FDA, and then future positive results from the ongoing intrathecal clinical trials."

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About Cure SMA

Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA)—a disease that takes away a person's ability to walk, eat, or breathe. It is the number one genetic cause of death for infants.

Since 1984, we've directed and invested in comprehensive research that has shaped the scientific community's understanding of SMA. We are currently on the verge of breakthroughs that will strengthen bodies, extend life, and lead to a cure.

We have deep expertise in every aspect of SMA—from the day-to-day realities to the nuances of care options—and until we have a cure, we'll do everything we can to support and advocate for those affected by the disease.

Learn more about how you can help us reach a treatment and cure at www.cureSMA.org.