Biogen Secures European Approval for High Dose SPINRAZA® Treatment
Biogen Inc. (Nasdaq: BIIB) has announced that the European Commission (EC) has granted marketing authorization for a new high dose regimen of SPINRAZA® (nusinersen). This authorization includes doses of 50 mg/5 mL and 28 mg/5 mL, aimed at treating 5q spinal muscular atrophy (SMA), the most prevalent form of the disease which constitutes approximately 95% of all SMA cases.
Details of the High Dose Regimen
The new treatment protocol features a more expedited loading phase, which involves administering two 50 mg loading doses spaced 14 days apart, followed by 28 mg maintenance doses every four months. Patients transitioning from the previously approved 12 mg dose will receive a single 50 mg dose instead of their next 12 mg dose, followed by the new 28 mg regimen.
Clinical Evidence Supporting Approval
The EC's approval is supported by findings from the three-part, Phase 2/3 DEVOTE study, which demonstrated significant efficacy of the high dose regimen. Treatment-naïve infants receiving the high-dose regimen showed statistically significant gains in motor function. Specifically, results indicated a mean difference of 26.19 points on the CHOP-INTEND scale when compared to a matched sham group from the ENDEAR study (p<0.0001).
- 26.19 points mean improvement on CHOP-INTEND
- 15.1 vs. -11.1 in treated vs. sham groups
- Mean improvement of 1.8 points on the Hammersmith Functional Motor Scale – Expanded
Statements from Biogen Leadership
Dr. Priya Singhal, Executive Vice President and Head of Development at Biogen, stated, “Since its approval in the European Union in 2017, SPINRAZA has set a new standard in patient care, successfully treating more than 10,000 individuals worldwide. We are committed to delivering this high dose regimen to the European SMA community as swiftly as possible.”
Safety Profile and Tolerability
The high dose regimen was generally well tolerated throughout the DEVOTE study, with adverse events aligning with those typically associated with SMA and the established safety profile of nusinersen. No new safety concerns were identified during the ongoing long-term extension study, and the most common adverse events included pneumonia and malnutrition.
Global Regulatory Status
The updated dosing regimen of SPINRAZA is already approved in Japan and is currently under review by the U.S. Food and Drug Administration (FDA), with a decision anticipated by April 3, 2026. Biogen continues to collaborate with regulatory bodies globally to enhance treatment accessibility for patients living with SMA.
About SPINRAZA and Its Impact
SPINRAZA is designed for intrathecal use via lumbar puncture performed by experienced healthcare professionals. This innovative treatment option is part of Biogen's ongoing commitment to improving outcomes in SMA care.
For additional information regarding SPINRAZA, including important safety details and prescribing information, prospective users can visit the product website as applicable to their region.