AveXis Inc, a Novartis company, has received FDA approval for Zolgensma (onasemnogene abeparvovec-xioi), reportedly the first gene therapy approved to treat children less than 2 years of age with spinal muscular atrophy (SMA).

Zolgensma received Fast Track, Breakthrough Therapy, Priority Review, and Orphan Drug designations. AveXis was also awarded a rare pediatric disease priority review voucher, under a program intended to encourage the development of new drugs and biological products for the prevention and treatment of certain rare pediatric diseases, according to a media release from the US Food and Drug Administration.

SMA is a rare genetic disease caused by a mutation in the survival motor neuron 1 (SMN1) gene. It is generally classified into several subtypes, based on the age of onset and severity; infantile-onset SMA is the most severe and most common subtype. Children with this condition have problems holding their head up, swallowing and breathing.

“Children with SMA experience difficulty performing essential functions of life. Most children with this disease do not survive past early childhood due to respiratory failure,” says Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in the release.

Zolgensma is an adeno-associated virus vector-based gene therapy that targets the cause of SMA. A one-time intravenous administration of Zolgensma delivers a fully functional copy of human SMN gene into the target motor neuron cells, which improves muscle movement and function, and survival of a child with SMA. Dosing is determined based on the weight of the patient.

The safety and effectiveness of Zolgensma is based on an ongoing clinical trial and a completed clinical trial involving a total of 36 pediatric patients with infantile-onset SMA between the ages of approximately 2 weeks and 8 months at study entry. The primary evidence of effectiveness is based on results from the 21 patients treated with Zolgensma in the ongoing clinical trial.

In this trial, there are 19 remaining patients, who range in age from 9.4 to 18.5 months; 13 of these 19 patients are at least 14 months of age. Compared to the natural history of patients with infantile-onset SMA, patients treated with Zolgensma also demonstrated significant improvement in their ability to reach developmental motor milestones (eg, head control and the ability to sit without support), the release explains.

“A diagnosis of SMA is devastating, leaving untreated babies who have the most severe form with painfully short, highly medicalized lives, during which they are unable to lift their heads, sit or roll, have difficulty swallowing and breathing and need 24-hour care,” says Jerry Mendell, MD, principal investigator at the Center for Gene Therapy at The Abigail Wexner Research Institute of Nationwide Children’s Hospital in Columbus, OH, in a separate news release from AveXis.

“In the START clinical trial we conducted with Zolgensma, all children were alive at the conclusion of the study and many were able to sit, roll, crawl, play and some could walk,” he shares.

“We are grateful to the tenacious researchers, partners and families who participated in the Zolgensma clinical trials that helped us achieve this incredible milestone,” states Dave Lennon, president of AveXis.

“We are proud to bring this one-time gene therapy to pediatric patients with SMA and remain committed to advancing the science behind Zolgensma to transform SMA, as well as other rare genetic diseases.”

“Zolgensma is poised to be another life-altering therapy for the SMA community,” comments Lynn O’Connor Vos, president and CEO of the Muscular Dystrophy Association, in a third media release, from MDA.

“It represents a breakthrough toward the promise of safe and effective gene therapies, and it may catalyze the development of other gene therapies to treat a range of rare neuromuscular diseases,” she adds.

[Source(s): US Food and Drug Administration, AveXis Inc, Muscular Dystrophy Association, PR Newswire]