Biogen upbeat on tofersen for SOD1-ALS despite mixed AdCom vote

US biotech Biogen (Nasdaq: BIIB) is focusing on the positives after a US Food and Drug Administration (FDA) advisory committee (AdCom) meeting on tofersen, an investigational product for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).

The Peripheral and Central Nervous System Drugs AdCom considered Biogen’s New Drug Application (NDA) for tofersen for the treatment of SOD1-ALS, which was submitted to the FDA for consideration under accelerated approval.

This was despite a Phase III trial having failed to meet its primary endpoint, though Biogen argued that the study and its open-label extension showed signs of slowing disease progression in people with SOD1-ALS. Trends favoring tofersen were seen across multiple secondary and exploratory measures of biologic activity and clinical function.