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MDA CELEBRATES FDA APPROVAL OF ZOLGENSMA FOR TREATMENT OF SPINAL MUSCULAR ATROPHY: The Muscular Dystrophy Association (MDA) celebrated on May 24th the decision by the U.S. Food and Drug Administration (FDA) to grant approval of Zolgensma (onasemnogene abeparvovac-xioi), the first gene therapy for a neuromuscular disease.

Zolgensma is a one-time intravenous (into the vein) infusion for the treatment of pediatric patients less than two years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene, including those who are pre-symptomatic at diagnosis.

SMA is the leading genetic cause of infant death. Zolgensma is designed to target the genetic root cause of SMA by delivering the survival motor neuron gene (SMN), which is missing or mutated in SMA. Zolgensma will be made available in the United Statesand will be marketed by AveXis, a Novartis company.

Approval of the therapy marks another historic achievement for the SMA community. Now, in addition to Spinraza--the first SMA disease-modifying therapy, which was approved in December 2016 for SMA--patients will have access to another promising therapy.

For decades, MDA has funded research aimed at the discovery of genes causing neuromuscular disease and has supported work to develop therapies that address the root cause of disease. Zolgensma is only the second gene therapy approved by the FDA to treat any disease, placing the field of neuromuscular disease at the forefront of genetic medicine. "Zolgensma is poised to be another life-altering therapy for the SMA community," says MDA President and CEO Lynn O'Connor Vos. "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."

Zolgensma may be the first of many gene therapies targeting the genetic root cause of disease, highlighting the importance of years of investment by MDA and others into gene identification and research unlocking the cause of disease. In addition, MDA has funded landmark research focused on developing and refining gene delivery tools and has supported the work to establish protocols for safe and effective gene therapy clinical trials. While this may be the first gene therapy for treating neuromuscular disease, the increasing pace of drug development holds immense promise for the future of the field. In the past decade alone, seven therapies for treating neuromuscular diseases have been approved by the FDA.

The Muscular Dystrophy Association is committed to transforming the lives of people affected by muscular dystrophy, ALS, and related neuromuscular diseases. This is done through innovations in science and innovations in care. For more information visit

Caption: LIFE-ALTERING: Zolgensma is designed to address the genetic root cause of SMA by replacing the defective or missing SMN1 gene to halt disease progression with a single, one-time infusion.

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Title Annotation:WHAT'S HAPPENING
Publication:The Exceptional Parent
Date:Jun 1, 2019
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