First Ever Gene Therapy for Spinal Muscular Atrophy Approved by the FDA

According to a story from PR Newswire, the Novartis company AveXis recently announced that the US Food and Drug Administration (FDA) has approved Zolgensma, making it the first ever gene therapy to be approved for the treatment of spinal muscular atrophy, a rare disease that is often fatal without prompt treatment. The single use therapy is designated for use in patients that are less than two years old who possess bi-allelic mutations of the SMN1 gene. The approval of Zolgensma will revolutionize the treatment of spinal muscular atrophy.

About Spinal Muscular Atrophy (SMA)

Spinal muscular atrophy is a type of neuromuscular disorder in which the motor neurons are destroyed, leading to muscle wasting. In many cases, the disease is lethal. This disorder is linked to genetic defects of the SMN1 gene. This gene encodes a protein called SMN, and when not present in certain amounts, neurons are unable to function. There are different kinds of spinal muscular atrophy that are categorized by when symptoms first appear. These symptoms may include loss of reflexes, muscle weakness and poor muscle tone, problems with feeding and swallowing, developmental delays, respiratory muscle weakness, tongue twitching, and a bell shaped torso. The most effective treatment currently available for the disease is called Spinraza. To learn more about spinal muscular atrophy, click here.

A Treatment Breakthrough

The approval of this gene therapy is a testament to the potential of this approach to treating genetic disorders like spinal muscular atrophy. A decade ago, there was little chance of long term survival for patients with severe forms of the disorder, but Zolgensma, which is administered only once, is able to provide long term benefit and allow patients to develop normally.

A clinical trial of the therapy saw no decrease in treatment benefit after four years, and all patients that received the treatment survived and were able to obtain levels of movement and development far beyond the natural scope of the disease progression. While it may not be appropriate to call Zolgensma a complete cure, it is absolutely a treatment breakthrough for spinal muscular atrophy. With this approval, patients born with the disorder can live practically symptom-free with prompt treatment.


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