Novartis’ SMA Gene Therapy Is on the Fast Track for Approval


By Alex Keown


Novartis’ gene therapy treatment for spinal muscular atrophy could see approval as early as May of next year after the U.S. Food and Drug Administration (FDA) granted AVXS-101, now dubbed Zolgensma, priority review.

The FDA accepted the company’s Biologics License Application. It had already earned Breakthrough Therapy designation. If approved, Zolgensma is on track to be one of the most expensive medications on the market. The drug, a one-time treatment for the fatal disease, is expected to have a price tag of $4 million to $5 million, Reuters reported this morning. The one-time cost is expected to outweigh the long-term costs of caring for SMA patients over a 10-year period, which can hit more than $10 million.

Novartis gained Zolgensma earlier this year after it acquired Illinois-based AveXis for $8.7 billion. SMA is caused by a defective or missing gene known as SMN1. Without this gene, infants with Type 1 SMA quickly lose the motor neurons that are responsible for basic muscle functions such as breathing, swallowing, speaking and walking. If untreated, it eventually leads to paralysis or death, typically by the age of 24 months. Zolgensma is a one-time infusion that replaces the missing or defective SMN1 gene with a functional copy. The new gene makes SMN protein, which improves motor neuron function in the infants and their likelihood of survival. Currently, there is only one other SMA treatment on the market, Biogen’s Spinraza, which was approved in December 2016.

David Lennon, president of AveXis, said the FDA’s granting of priority review to Zolgensma brings the company closer to providing a treatment for SMA Type 1.

“As a one-time infusion that addresses the genetic root cause of SMA without the need for repeat dosing, Zolgensma represents a potentially significant therapeutic advance for these patients and their families,” Lennon said in a statement. “The introduction of one-time, potentially curative therapies will require rethinking how our healthcare system manages diagnosis, treatment, care and associated costs for patients with genetic disease.”

Lennon added that Novartis and AveXis are committed to “flexibly partnering with healthcare stakeholders to ensure appropriate access to our medicines.”

Clinical data shows the 15 patients who were infused with Zolgensma were all able to breathe without assistance from a permanent ventilator. Additionally, 92 percent (11 of 15) were able to sit unassisted for more than five seconds, which AveXis said is a never-before-achieved milestone in the natural history of SMA Type 1. Natural history indicates that more than 90 percent of untreated patients with SMA Type 1 will die or require permanent ventilation by 24 months of age, the company said.

In addition to the SMA treatment, AveXis has a rare disease pipeline of gene therapy assets for Rett Syndrome and a genetic form of amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 (SOD1) gene.



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