EMA Panel Backs High-Dose SPINRAZA Regimen for Spinal Muscular Atrophy

Biogen announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a unanimous positive opinion recommending approval of a high-dose regimen of nusinersen (SPINRAZA®) for the treatment of 5q spinal muscular atrophy (SMA), the most common form of the disease. The European Commission is expected to make a final decision by January 2026. If approved, the new regimen would complement the currently authorized 12 mg dose.

Biogen said the recommendation marks an important step toward expanding treatment options for SMA—an area where unmet needs remain despite significant advances. “The CHMP’s positive opinion represents a promising advancement in our commitment to support the evolving needs of individuals living with SMA,” said Dr. Priya Singhal, Biogen’s Head of Development.

SPINRAZA is currently approved in more than 71 countries at the standard 12 mg dose. The high-dose regimen was evaluated in the Phase 2/3 DEVOTE study and its ongoing extension, involving both treatment-naïve patients and individuals previously treated with the 12 mg dose.

The new regimen consists of two 50 mg loading doses given 14 days apart, followed by 28 mg maintenance doses every four months. Patients transitioning from the 12 mg dose receive one 50 mg loading dose before moving to maintenance dosing. All doses are administered intrathecally by trained healthcare professionals.

Results from DEVOTE showed significant clinical benefit. In the pivotal Part B cohort of 75 infants, treatment-naïve patients receiving high-dose nusinersen demonstrated statistically significant improvement in motor function compared with a matched untreated group from the earlier ENDEAR study. Patients also experienced a 68% reduction in the risk of death or permanent ventilation. In Part C, participants who switched to the high-dose regimen after nearly four years on the standard dose showed measurable gains in motor function.

Investigators welcomed the findings as a major advance. “Based on the results from the DEVOTE study and my experience with patients, high-dose nusinersen has the potential to bring meaningful benefits to people living with SMA,” said Professor Eugenio Mercuri, a leading pediatric neurologist.

Safety findings were consistent with the known profile of SPINRAZA and the underlying disease, with no new concerns observed in long-term follow-up.

The high-dose regimen was recently approved in Japan and is currently under review by the U.S. FDA, with a decision expected in April 2026. Biogen said it continues working with global regulators to make the new dosing option available to SMA patients worldwide.