First oral treatment for spinal muscular atrophy nets FDA approval
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The FDA approved risdiplam for patients aged at least 2 months with spinal muscular atrophy, according to an agency press release.
Risdiplam (Evrysdi, Genentech) is the first oral drug approved to treat spinal muscular atrophy.
“Evrysdi is the first drug for this disease that can be taken orally, providing an important treatment option for patients with [spinal muscular atrophy], following the approval of the first treatment for this devastating disease less than 4 years ago,” Billy Dunn, MD, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, said in the release.
Risdiplam was evaluated for the treatment of infantile-onset and later-onset spinal muscular atrophy in two clinical studies.
An open-label study based efficacy on the ability of patients (mean age, 6.7 months) to sit upright without support for at least 5 seconds in addition to survival without permanent ventilation. At 12 months of treatment, 41% of patients were able to sit upright without additional support, and after 23 or more months, 81% lived without the need for permanent ventilation, according to the release.
Treatment of later-onset spinal muscular atrophy was evaluated in a randomized, placebo-controlled study, with the primary endpoint of change in motor function from baseline to 1 year (MFM32 score). At 1 year, patients who received risdiplam experienced an average MFM32 score increase of 1.36, compared with a 0.19 decrease in the placebo group.
According to the release, common side effects of risdiplam include fever, diarrhea, rash, ulcers of the mouth area, joint pain and urinary tract infections.
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