The U.S. Food and Drug Administration (FDA) has announced a three-month extension for reviewing Biogen's tofersen, an investigational treatment for amyotrophic lateral sclerosis (ALS) patients with mutations in the superoxide dismutase 1 (SOD1) gene. The regulatory decision, originally expected in January, has been pushed to April following what the FDA termed a "major amendment" to the filing.
The delay stems from Biogen's responses to FDA queries, though specific details of these information requests remain undisclosed. Priya Singhal, Biogen's global head of safety and regulatory sciences, emphasized the company's commitment to providing all necessary information to facilitate the FDA's review process.
Clinical Development and Trial Data
Tofersen, developed in partnership with Ionis Pharmaceuticals, is an antisense oligonucleotide specifically targeting SOD1-ALS, a rare form affecting approximately 2% of ALS cases. The therapy entered FDA review in July under the accelerated approval pathway with priority review status, based on data from the VALOR trial.
While initial VALOR trial results were disappointing, subsequent data demonstrated the drug's potential to slow disease progression in this devastating neurodegenerative condition. A confirmatory phase 3 trial, ATLAS, is currently ongoing with results expected in 2026.
Treatment Access and Market Context
During the extended review period, Biogen will continue providing tofersen through its early-access program, maintaining availability for eligible patients. The FDA has not yet announced whether an advisory committee meeting will be scheduled to review the drug's data.
The therapy could address a significant unmet need in the ALS community, which affects approximately 31,000 patients in the United States. For Biogen, tofersen represents an important component of their pipeline strategy, alongside other key developments including the Alzheimer's candidate lecanemab, antidepressant zuranolone, and lupus therapies litifilimab and dapirolizumab pegol.
Regulatory Implications
The FDA's decision to extend the review period highlights the complexity of evaluating novel therapies for rare genetic conditions. The agency will need to determine whether the available VALOR trial data sufficiently supports conditional approval while awaiting results from the confirmatory ATLAS study.
This regulatory extension comes at a crucial time for Biogen, as the company faces competitive pressures in its multiple sclerosis and spinal muscular atrophy portfolios, while simultaneously working to establish new revenue streams following the limited commercial success of its Alzheimer's therapy Aduhelm (aducanumab).
