Update on FDA Advisory Committee meeting on tofersen for SOD1-ALS.- Biogen.
Biogen Inc. announced the outcome of the FDA's Peripheral and Central Nervous System Drugs Advisory Committee meeting on tofersen, an investigational product for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).
On the question, “Is the available evidence sufficient to conclude that a reduction in plasma neurofilament light chain (NfL) concentration in tofersen-treated patients is reasonably likely to predict clinical benefit of tofersen for treatment of patients with SOD1-ALS?” the Committee voted unanimously yes (9 yes to 0 no), for consideration of a potential accelerated approval.
On the second question, “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?” 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.
Estimates hold that less than 500 of the roughly 30,000 ALS patients in the U.S. have this form of the neurodegenerative disease. As it is hereditary, SOD1 ALS often passes from generation to generation, affecting many members of the same family.
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