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Biogen’s Salanersen Granted FDA Breakthrough Therapy Designation for Spinal Muscular Atrophy
Friday, June 05, 2026
Biogen announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to salanersen for the treatment of spinal muscular atrophy (SMA).
Salanersen is an investigational antisense oligonucleotide (ASO) therapy designed to provide high efficacy with once-yearly dosing. The Breakthrough Therapy Designation is intended to speed up the development and review of medicines that may offer significant improvements over existing treatments for serious conditions.
The designation was supported by data from a Phase 1b study presented at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference and the 5th International Scientific Congress on SMA. The study evaluated children with SMA who had shown a suboptimal response to previous gene therapy.
Results showed clinically meaningful improvements in motor function after treatment with salanersen. The therapy was also associated with reduced neurofilament levels, indicating a slowing of neurodegeneration. Salanersen was generally well tolerated during the study.
Biogen is advancing salanersen through a global Phase 3 development programme comprising three studies.
STELLAR-1, which is currently recruiting, is an open-label study assessing salanersen in treatment-naïve, clinically presymptomatic infants under six weeks of age who have a genetic diagnosis of SMA.
SOLAR, also recruiting participants, is evaluating salanersen in adolescents and adults aged 15 to 60 years with SMA who are either treatment-naïve or have previously received risdiplam.
STELLAR-2, expected to begin recruitment in June 2026, is a randomised, double-blind, sham-controlled study. It will assess salanersen in infants with SMA who received presymptomatic gene therapy treatment at six weeks of age or younger, with salanersen initiated approximately six months after gene therapy.
The FDA designation marks an important regulatory milestone for Biogen as it continues to develop salanersen as a potential new treatment option for people living with SMA.
Source: globenewswire.com
