Biogen Receives European Commission Approval for High Dose Regimen of SPINRAZA® (nusinersen) for Spinal Muscular Atrophy

Core Viewpoint - Biogen Inc. has received marketing authorization from the European Commission for a high dose regimen of SPINRAZA (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA), which is the most common form of the disease, accounting for approximately 95% of all SMA cases [1][2] Product Authorization and Dosing Regimen - The updated SPINRAZA regimen includes 50 mg/5 mL and 28 mg/5 mL doses, featuring a more rapid loading phase with two 50 mg doses administered 14 days apart, followed by 28 mg maintenance doses every four months [1][11] - Individuals transitioning from the previous 12 mg dose will receive one 50 mg dose instead of their next 12 mg dose, followed by the 28 mg maintenance doses [1] Clinical Study and Efficacy - The European Commission's approval is based on data from the Phase 2/3 DEVOTE study, which demonstrated statistically significant improvements in motor function for treatment-naïve, symptomatic infants using the high dose regimen compared to a matched sham group [2][6] - The pivotal cohort showed a mean difference of 26.19 points on the CHOP-INTEND scale, with a significant p-value of <0.0001 [2][9] - Participants transitioning from the low dose regimen also experienced a mean improvement of 1.8 points on the Hammersmith Functional Motor Scale – Expanded from baseline to Day 302 [2] Safety Profile - The high dose regimen was generally well tolerated, with adverse events consistent with SMA and the known safety profile of nusinersen, with no new safety concerns observed [3][6] - Common adverse events reported in at least 10% of participants included pneumonia, COVID-19, pneumonia aspiration, and malnutrition [3] Community and Regulatory Engagement - Biogen is committed to advancing care for individuals living with SMA and is working with regulatory authorities globally to progress the high dose regimen, which is also approved in Japan and under review by the U.S. FDA [7][11] - The approval highlights the importance of sustained research and investment in SMA care, as noted by community leaders [5]