Today, Biogen announced the publication of results from the pivotal Phase 2/3 DEVOTE study in Nature Medicine, highlighting the potential benefits of a high-dose regimen of nusinersen for people living with spinal muscular atrophy (SMA). The study evaluated a higher loading and maintenance dose regimen of nusinersen and demonstrated improvements across key areas, including motor function, bulbar function, respiratory health, and survival.
Findings from the DEVOTE study showed that the high-dose regimen led to faster reductions in neurodegeneration, measured by neurofilament levels, compared to the currently approved 12 mg regimen. In treatment-naïve infants, the high-dose regimen resulted in statistically significant improvements in motor function compared to untreated infants from a matched historical study. Participants who transitioned from the 12 mg regimen to the high-dose regimen also experienced gains in motor and upper limb function.
The DEVOTE study enrolled 139 participants across a wide range of ages, SMA types, and prior treatment experience. The safety profile of the high-dose regimen was generally consistent with the known safety profile of the 12 mg regimen.
The high-dose regimen of nusinersen is currently approved in the European Union and Japan. In the United States, it is under review by the Food and Drug Administration (FDA), with a Prescription Drug User Fee Act (PDUFA) action date of April 3, 2026.
For more information or to learn more about this clinical trial, please contact Biogen at [email protected] or call the Biogen Clinical Trials Center at 866-633-4636.
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