Biogen has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending approval of the high dose regimen of nusinersen for the treatment of 5q spinal muscular atrophy (SMA).
A final decision from the European Commission is expected in January 2026.
Priya Singhal, Executive Vice President and Head of Development at Biogen, said: “While we’ve seen great progress over the past decade, there is urgency to do more to address the clear unmet needs of the SMA community.
“The CHMP’s positive opinion for the high dose regimen of nusinersen represents a promising advancement in our commitment to support the evolving needs of individuals living with SMA and deliver therapies that can enhance patient outcomes.”
The recommendation is based on data from the phase 2/3 DEVOTE study and its ongoing long-term extension.
The study evaluated the high dose regimen in treatment-naive participants and those transitioning from the approved 12 mg dose. The new regimen includes a loading phase of two 50 mg doses 14 days apart, followed by maintenance doses of 28 mg every four months.
Eugenio Mercuri, Professor of Pediatric Neurology at the Catholic University in Rome, explained: “The positive CHMP opinion for the high dose regimen of nusinersen is an important milestone for the SMA community.
“Based on the results from the DEVOTE study and my experience with patients receiving this novel regimen, I am confident that high dose nusinersen has the potential to bring meaningful benefits to people living with SMA.”
The high dose regimen was generally well tolerated, with no new safety concerns observed. Biogen is working with global regulatory authorities to advance the regimen as an additional option for people living with SMA.
