- Biogen's updated data on nusinersen (SPINRAZA®) shows significant improvements in motor function for SMA patients.
- The higher dose regimen exhibits potential for enhanced outcomes, particularly in non-ambulatory patients, while ensuring safety.
- The global regulatory review is ongoing for the new dosing regimen of nusinersen.
Biogen (BIIB, Financial) has announced new clinical data from its DEVOTE Part C study, revealing that transitioning patients with spinal muscular atrophy (SMA) from the standard 12 mg SPINRAZA dose to a higher dose regimen led to functional improvements. The new dosing involves two initial 50 mg loading doses, followed by 28 mg maintenance doses every four months. Non-ambulatory participants showed a notable improvement of +2.5 on the HFMSE scale, suggesting significant therapeutic benefits. The safety profile remained consistent with existing data for the 12 mg dosage.
Further reinforcing the importance of early intervention, the final results from the NURTURE study demonstrated that 92% of presymptomatic infants achieved independent walking capabilities, with all participants surviving and maintaining clinical benefits over eight years. The study highlighted the pivotal role of early treatment in SMA, emphasizing that no participants required permanent ventilation, underscoring the profound long-term benefits of early nusinersen therapy.
These findings, presented at the SMA Research & Clinical Care Meeting by Cure SMA, could potentially reshape clinical practices for SMA treatment. Biogen's applications for this higher dose regimen are currently under review across multiple major markets, including the United States, Europe, and Japan.
