Biogen’s Salanersen Receives FDA Breakthrough Therapy Designation for Spinal Muscular Atrophy
Friday, June 05, 2026
Biogen has announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to salanersen for the treatment of spinal muscular atrophy (SMA).
The designation is intended to accelerate the development and review of medicines for serious conditions when early clinical evidence suggests they may offer significant improvements over existing treatment options. Salanersen is an investigational antisense oligonucleotide (ASO) therapy that is being developed as a once-yearly treatment for SMA.
The FDA’s decision was based on results from the Phase 1b study of salanersen, which were recently presented at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference and SMA Europe 2026. In the study, children with SMA who had shown a suboptimal response to previous gene therapy experienced clinically meaningful improvements in motor function after receiving salanersen. The treatment was also associated with reduced neurofilament levels, indicating a slowing of neurodegeneration, and was generally well tolerated.
Biogen stated that the designation highlights the continued need for improved treatment options for people living with SMA and supports the potential of salanersen to provide meaningful benefits beyond currently available therapies.
The company is advancing a Phase 3 clinical programme comprising three global studies. STELLAR-1, which is currently recruiting, is evaluating salanersen in treatment-naïve, clinically presymptomatic infants under six weeks of age with a genetic diagnosis of SMA. SOLAR, also recruiting, is assessing the therapy in adolescents and adults aged 15 to 60 years who are either treatment-naïve or have previously received risdiplam.
A third study, STELLAR-2, is expected to begin recruitment in June 2026. The randomised, double-blind, sham-controlled trial will evaluate salanersen in infants with SMA who received presymptomatic gene therapy treatment with onasemnogene abeparvovec-xioi at six weeks of age or younger, with salanersen initiated approximately six months later.
Biogen said the Breakthrough Therapy Designation marks an important milestone for its SMA development programme as it continues to evaluate the potential role of salanersen in future treatment approaches for the disease.
Source: globenewswire.com
