USFDAJun 4, 2026, 07:31 AM
Biogen's Salanersen Gets FDA Breakthrough Therapy Designation for SMA
AI Summary
Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has granted salanersen Breakthrough Therapy Designation for the treatment of spinal muscular atrophy (SMA). This designation aims to expedite the development and review of the investigational once-yearly antisense oligonucleotide (ASO), which has shown potential for high efficacy. The decision is based on Phase 1b study data, where children with SMA previously treated with gene therapy experienced clinically meaningful improvements in motor function and slowing of neurodegeneration after initiating salanersen. The company is advancing salanersen into three global Phase 3 studies.
Key Highlights
- FDA granted salanersen Breakthrough Therapy Designation for spinal muscular atrophy (SMA).
- Designation is supported by Phase 1b data showing improvements in motor function and slowing neurodegeneration.
- Salanersen is an investigational once-yearly antisense oligonucleotide (ASO) for SMA.
- Phase 1b study showed meaningful 75% reductions in neurofilament light chain (NfL) levels.
- 12 of 24 participants in Phase 1b achieved at least one new WHO motor milestone.
- Salanersen was generally well-tolerated in the Phase 1b study at 40 mg and 80 mg doses.
- Three global Phase 3 studies (STELLAR-1, SOLAR, STELLAR-2) are underway or recruiting.
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