The European Commission has approved a higher-dose SPINRAZA (nusinersen) regimen for spinal muscular atrophy in the EU, supported by DEVOTE study data showing improved motor outcomes with a consistent safety profile.
Written By: Pharmacally Medical News Desk
The European Commission has granted marketing authorization for a new high-dose regimen of SPINRAZA (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA), marking an important update to the therapy’s approved use across the European Union. The decision expands the existing EU label for SPINRAZA and introduces a revised dosing strategy designed to deliver higher drug exposure earlier in treatment. SPINRAZA is developed and commercialized by Biogen and has been a foundational therapy in SMA care since its initial EU approval in 2017.
What the new high-dose regimen includes
Under the updated authorization, the high-dose SPINRAZA regimen consists of:
- Two loading doses of 50 mg administered 14 days apart
- Follow-on maintenance doses of 28 mg given once every four months
Patients currently receiving the original 12 mg regimen can transition by replacing their next scheduled 12 mg dose with a single 50 mg dose, followed by ongoing 28 mg maintenance dosing. As with the original formulation, SPINRAZA is administered intrathecally via lumbar puncture by trained healthcare professionals.
The approval applies to infants, children, adolescents, and adults with genetically confirmed 5q SMA, which accounts for approximately 95 percent of all SMA cases.
Clinical evidence supporting approval
The European Commission’s decision was based on data from the Phase 2/3 DEVOTE study (NCT04089566) and its ongoing long-term extension. DEVOTE was a three-part, global, dose-escalation trial that evaluated the safety, pharmacokinetics, and efficacy of higher doses of nusinersen across a broad SMA population.
In the pivotal cohort of treatment-naïve, symptomatic infants with SMA type 1, the high-dose regimen demonstrated statistically significant improvements in motor function compared with a prespecified matched untreated control group from the ENDEAR study. Motor function was assessed using the CHOP-INTEND scale, where treated infants showed a mean improvement of 15.1 points versus a decline of 11.1 points in the control group, resulting in a mean treatment difference of 26.19 points.
Additional benefits were observed in older children and adults who transitioned from the 12 mg regimen to the higher dose. In this open-label cohort, patients experienced a mean improvement of 1.8 points on the Hammersmith Functional Motor Scale–Expanded by Day 302, indicating functional gains across a wider age range and disease spectrum.
Safety profile and tolerability
Across DEVOTE and its extension, the high-dose SPINRAZA regimen was generally well tolerated, with a safety profile consistent with long-term experience from the standard 12 mg dose. No new safety signals were identified with continued higher-dose use.
The most frequently reported adverse events, occurring in at least 10 percent of participants and more often than in untreated controls, included pneumonia, COVID-19, aspiration pneumonia, and malnutrition. Known precautions associated with nusinersen remain unchanged and include risks related to lumbar puncture procedures, thrombocytopenia and coagulation abnormalities, renal toxicity, and hydrocephalus.
Nicole Gusset, CEO of SMA Europe said as a patient advocacy community we have welcomed the decision, highlighting that additional dosing options support more individualized treatment approaches as the SMA therapeutic landscape continues to mature.
In addition to the European Union, the high-dose SPINRAZA regimen is already approved in Japan. In the United States, the regimen remains under review by the FDA, with a regulatory decision expected by April 3, 2026. Biogen has indicated that discussions with other global regulatory authorities are ongoing to broaden access to this dosing option.
References
Biogen Receives European Commission Approval for High Dose Regimen of SPINRAZA® (nusinersen) for Spinal Muscular Atrophy, 12 January 2026, https://investors.biogen.com/node/30071/pdf
Study of Nusinersen (BIIB058) in Participants with Spinal Muscular Atrophy (DEVOTE), ClinicalTrials.gov ID NCT04089566, https://www.clinicaltrials.gov/study/NCT04089566
Finkel RS et al, DEVOTE Study Exploring Higher Dose of Nusinersen in Spinal Muscular Atrophy: Study Design and Part A Results. J Neuromuscul Dis. 2023;10(5):813-823. PMID: 37393513; PMCID: PMC10578235, https://doi.org/10.3233/jnd-221667