A New Drug for a Serious Disorder

FDA OKs new treatment for rare Duchenne muscular dystrophy mutation

(RxWiki News) The US Food and Drug Administration (FDA) has approved a new targeted treatment for a rare Duchenne muscular dystrophy (DMD) mutation.

The agency approved Viltepso (viltolarsen) injection to treat DMD in those who have a mutation of a particular gene. Roughly 8 percent of people who have DMD have the gene mutation that this newly approved drug targets.

This is the second drug approved to treat this DMD mutation, according to the FDA.

“The FDA is committed to fostering drug development for serious neurological disorders like Duchenne muscular dystrophy,” said Dr. Billy Dunn, director of the FDA's Office of Neuroscience, in a press release. “Today’s approval of Viltepso provides an important treatment option for Duchenne muscular dystrophy patients with this confirmed mutation.”

In DMD, gene mutations cause an absence of dystrophin. This is a protein that preserves muscle cells. The absence of this protein leads to ongoing muscle deterioration and weakness.

Two studies that included a total of 32 patients with DMD assessed the effectiveness and safety of Viltepso. This drug appeared to drastically increase the amount of dystrophin in those who received it.

An increase in dystrophin is likely to result in benefits for those who have DMD, the FDA noted. But the studies of this drug did not establish a clinical benefit for Viltepso.

Because the FDA allowed this drug to be approved through an accelerated process, the agency is now requiring the drugmaker to conduct an additional study to confirm the supposed clinical benefit of Viltepso.

Common side effects of Viltepso included fever, cough, injection-site reactions and infections of the upper respiratory tract. Although the studies of this drug did not reveal kidney toxicity in any participants, the FDA noted that similar drugs have resulted in this condition. Because kidney toxicity can be fatal, the FDA said patients who take Viltepso should have their kidney function monitored.

The FDA granted approval of this drug to NS Pharma, Inc.

If you have questions about DMD or DMD treatments, speak with your health care provider.

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Review Date: 
September 4, 2020