"We are committed to supporting individuals with SMA and their families by advancing research that aims to answer critical questions for the community."
Today, Biogen announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) and the European Medicines Agency (EMA) has validated the application for a higher dose regimen of nusinersen for spinal muscular atrophy (SMA).
The FDA filing submission included data from the Phase 2/3 DEVOTE study, a three-part study that enrolled 145 patients across all ages and SMA types:
- Part A: an open-label safety evaluation period in children and teens (n=6) with later-onset SMA.
- Part B: a cohort with infantile onset-symptoms (n=75) and a later-onset cohort (n=24).
- Part C: an open-label evaluation in children and adults who transitioned from the approved dose of 12 mg to the 50/28 mg regimen (n=40).
Biogen also released an SMA Community Statement that provided a regulatory update. Click this link to learn more.
Next Steps
Cure SMA will keep the community updated on news as this application works its way through the regulatory process, which can take up to 10 months, and toward potential approval.
In August 2024, Cure SMA hosted a webinar for the community that reviewed the important steps that will follow positive results from clinical trials that could lead to regulatory filing and potentially next treatment approvals. Click here to see a recording.