FDA advisory committee votes for potential accelerated approval of tofersen for SOD1-ALS

The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted unanimously that tofersen, an antisense oligonucleotide for the treatment of superoxide dismutase 1 ALS, may be considered for potential accelerated approval.

According to a release from Biogen Inc, the committee ruled 9-0 that available evidence was sufficient to conclude that a reduction in plasma neurofilament light chain concentration in tofersen-treated patients was likely to predict clinical benefit for those with SOD1-ALS.

However, the committee split (3 yes, 5 no and 1 abstention) in its decision to consider traditional approval, based on whether clinical data from a placebo-controlled study, available long-term extension study results and additional data from the effects on relevant biomarkers provided substantial evidence of the effectiveness of tofersen.

The advisory committee, which provides nonbinding recommendations for FDA consideration, discussed both topics and agreed the benefit-risk profile was favorable, based on review of all data provided on tofersen for individuals with SOD1-ALS, the release stated.

“After hearing the moving experiences of the ALS community and reviewing the totality of data, the committee voted that reductions of neurofilament are reasonably likely to predict clinical benefit of tofersen,” Priya Singhal, MD, MPH, executive vice president, head of development and interim head of research and global safety and regulatory sciences at Biogen, said in the release. “If approved, tofersen would potentially represent a major advance for people living with SOD1-ALS.”

The FDA previously accepted a new drug application for tofersen and granted priority review for the drug in July 2022. A review of tofersen is ongoing, with a Prescription Drug User Fee Act action date of April 25, 2023.