First Oral Rx for Spinal Muscular Atrophy

FDA approves Evrysdi (risdiplam) to treat spinal muscular atrophy

(RxWiki News) The US Food and Drug Administration (FDA) has approved the first-ever oral medication for spinal muscular atrophy (SMA).

This new approval is for Evrysdi (risdiplam). The FDA approved it to treat patients 2 months of age and older.

SMA is a rare and often fatal disease that affects muscle strength and movement. SMA is genetic, meaning it is passed down through families.

“Evrysdi is the first drug for this disease that can be taken orally, providing an important treatment option for patients with SMA, following the approval of the first treatment for this devastating disease less than four years ago,” said Dr. Billy Dunn, director of the FDA's Office of Neuroscience, in a press release.

The effectiveness of risdiplam as a treatment for patients with infantile-onset and later-onset SMA was shown in two clinical studies.

For the infantile-onset study, efficacy was measured by the child's ability to sit without support for at least five seconds, as well as survival without permanent ventilation:

  • After 12 months of treatment, the studies showed that 41 percent of those who were given risdiplam were able to sit independently for more than five seconds.
  • After 23 or more months of treatment, 81 percent of those who were given risdiplam were alive without permanent ventilation.

The study for later-onset SMA included 180 patients. Those who were given risdiplam saw an increase in their total score on a test of motor function at the one-year mark.

Available as an oral solution, this medication is dosed for children by weight and age. For adults, the recommended dose is 5 mg per day.

This medication is to be taken/given after a meal and at approximately the same time each day. Risdiplam is not to be given with formula or milk.

Your child's health care provider will determine the amount of time for treatment.

Common side effects of risdiplam included fever, diarrhea, rash, ulcers of the mouth and joint pain. In the infantile-onset population, other side effects included constipation, vomiting, and lung and upper respiratory infections.

This new approval was granted to Genentech, Inc.