Biogen Updates On FDA Advisory Committee Meeting On Tofersen For SOD1-ALS

Biotechnology company Biogen Inc. (BIIB) announced the outcome of the U.S. Food and Drug Administration's Peripheral and Central Nervous System Drugs Advisory Committee meeting on tofersen, an investigational product to treat superoxide dismutase 1 or SOD1 amyotrophic lateral sclerosis or ALS.

Priya Singhal, Executive Vice President and Head of Development and interim Head of Research and Global Safety and Regulatory Sciences at Biogen stated that the Committee voted that reductions of neurofilament are reasonably likely to predict clinical benefit of tofersen. If approved, tofersen would potentially represent a major advance for people living with SOD1-ALS.

FDA Advisory Committees provide non-binding recommendations for consideration by the FDA. The New Drug Application for tofersen for the treatment of SOD1-ALS was submitted to the FDA for consideration under accelerated approval. The FDA is continuing its review of tofersen with a Prescription Drug User Fee Act action date of April 25, 2023.

Tofersen is an antisense oligonucleotide or ASO being evaluated as a treatment for SOD1-ALS, a rare genetic form of the disease affecting around 330 people in the United States.

In people with this form of the disease, mutations in their SOD1 gene cause their bodies to create a toxic form of SOD1 protein. This toxic protein causes motor neurons to degenerate, resulting in progressive muscle weakness. Tofersen is designed to bind to SOD1 mRNA and reduce SOD1 protein production.

In a statement, the company noted that the Committee voted unanimously yes with 9 votes for consideration of a potential accelerated approval on the question, "Is the available evidence sufficient to conclude that a reduction in plasma neurofilament light chain or NfL concentration in tofersen-treated patients is reasonably likely to predict clinical benefit of tofersen for treatment of patients with SOD1-ALS?"

The second question was "Does the clinical data from the placebo-controlled study and available long-term extension study results, with additional supporting results from the effects on relevant biomarkers (i.e., changes in plasma NfL concentration and/or reductions in SOD1), provide substantial evidence of the effectiveness of tofersen in the treatment of patients with SOD1-ALS?".

For this, the Committee voted 3 yes, 5 no and 1 abstain, for consideration of a potential traditional approval.

Additionally, the committee discussed both of these topics and reached consensus that the benefit-risk profile was favorable based on the review of the totality of data for tofersen in people with SOD1-ALS.

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