ARTICLE | Regulation

ALS panel to weigh accelerated vs. full approval of Biogen’s tofersen

FDA proposing two paths to regulatory flexibility; one would establish NfL as a surrogate endpoint for the disease

March 21, 2023 1:12 AM UTC

Both accelerated and full approval will be on the table at Wednesday’s FDA advisory committee meeting to discuss an application from Biogen for antisense therapy tofersen to treat amyotrophic lateral sclerosis patients with SOD1 mutations.

The agency previously announced that it was considering accelerated approval of tofersen based on neurofilament levels, but briefing documents released Monday, in advance of Wednesday’s panel on the therapy, indicate the agency is also open to granting full approval, despite tofersen’s clinical program producing no positive randomized-controlled trials. ...