FDA Approves Higher‑Dose Spinraza Regimen for Spinal Muscular Atrophy

Overview

• U.S. regulators cleared a high‑dose regimen of Spinraza (nusinersen) as a treatment option for people living with spinal muscular atrophy.
• The regimen uses an accelerated start with two 50 mg injections 14 days apart, then 28 mg maintenance injections every four months.
• FDA approval draws on the Phase 2/3 DEVOTE study, where treatment‑naïve symptomatic infants improved 26.19 points more than a matched sham group on the CHOP‑INTEND motor scale (p<0.0001).
• Biogen reports the safety profile at the higher dose was broadly in line with the established lower dose, with pneumonia, COVID‑19, aspiration pneumonia, and malnutrition more common in the infant cohort than in the historic control.
• Biogen says U.S. supply will reach clinics in the coming weeks, and Cure SMA plans an April 30 webinar to guide families on access and care decisions.