Biogen Gains European Commission Approval for High-Dose SPINRAZA® Regimen

New Dosing Option for Spinal Muscular Atrophy

Biogen has received European Commission approval for a high-dose regimen of SPINRAZA® (nusinersen), expanding treatment options for individuals living with 5q spinal muscular atrophy (SMA) — the most common form of the disease. The updated authorization revises the dosing schedule to include higher loading doses and a defined maintenance plan, providing clinicians with an alternative strategy to support patient care.

What the High-Dose Regimen Involves

The high-dose SPINRAZA regimen features two 50 mg loading doses administered 14 days apart, followed by 28 mg maintenance doses every four months thereafter. Patients transitioning from the standard 12 mg regimen will receive a single 50 mg dose before continuing with the 28 mg maintenance schedule.

Clinical Evidence Supporting Approval

The European Commission’s decision is grounded in data from the Phase 2/3 DEVOTE study and its long-term extension. Results showed that infants treated with the high-dose regimen achieved statistically significant improvements in motor function compared with matched historical controls. Additional analyses showed benefit in older children and adults who switched from the standard regimen, with overall improvements in functional motor scale measures.

Benefits and Safety Profile

The enhanced regimen demonstrated a generally consistent safety profile with the previously approved dose, supporting its use in diverse patient populations. Common adverse events observed in the study were aligned with the known safety characteristics of nusinersen and the SMA disease state. The high-dose option aims to help clinicians optimize treatment trajectories for people with SMA.

Impact on SMA Care

This approval marks an important regulatory milestone in the SMA landscape, reflecting ongoing efforts to refine therapeutic approaches and offer more tailored care. By expanding available dosing strategies, Biogen reinforces its commitment to advancing clinical options for communities affected by SMA and addressing unmet needs in neuromuscular disease management.