Overview
- CHMP issued a positive opinion recommending approval of a higher-dose regimen of nusinersen (Spinraza) for treating 5q spinal muscular atrophy.
- The proposed schedule uses two 50 mg loading doses 14 days apart followed by 28 mg maintenance doses every four months.
- The recommendation draws on phase II/III DEVOTE data and its extension in treatment‑naive patients and those switching from the approved 12 mg dose, with reported motor-function gains.
- Biogen reported the regimen was generally well tolerated with no new safety concerns identified.
- The opinion now goes to the European Commission for a final decision expected in January 2026, after a separate FDA rejection in September requested CMC updates.