FDA Approves Option for Spinal Muscular Atrophy

The US Food and Drug Administration has approved Evrysdi (risdiplam), an oral drug for the treatment of spinal muscular atrophy among patients aged 2 years or older.

The efficacy of Evrysdi was demonstrated in 2 clinical studies: one including patients with infantile-onset SMA (N = 21), and the other including patients with later-onset SMA (N = 180). Among patients with infantile-onset SMA, 41% of patients treated with Evrysdi for 12 months were able to sit independently for more than 5 seconds. After 23 months of treatment, 81% of patients stayed alive without the use of permanent ventilation.

In the second study, patients with later-onset SMA who were treated with Evrysdi experienced an average 1.36-point increase in Motor Function Measure-32 items (MFM32) score compared with an average increase of 0.19 with placebo, or inactive treatment.

Fever, rash, diarrhea, ulcers of the mouth area, urinary tract infections, and arthralgia are the most common adverse effects of Evrysdi. Adverse effects among patients with infantile-onset SMA were noted to be similar to those with later-onset SMA, with additional adverse effects of pneumonia, upper respiratory tract infection, vomiting, and constipation observed among patients with infantile-onset SMA.

—Christina Vogt

Reference:
FDA approves oral treatment for spinal muscular atrophy. News release. US Food and Drug Administration. August 7, 2020. Accessed August 10, 2020. https://www.fda.gov/news-events/press-announcements/fda-approves-oral-treatment-spinal-muscular-atrophy