FDA posts briefing document for advisors’ review of Biogen’s ALS drug

The FDA has posted to its website the briefing document for the Peripheral and Central Nervous System Drugs Advisory Committee, or PCNS, meeting being held on March 22 to discuss whether the observed reduction of neurofilament light chain is reasonably likely to predict clinical benefit and support accelerated approval of tofersen for the treatment of patients with Amyotrophic Lateral Sclerosis, or ALS, associated with mutations in the superoxide dismutase 1 gene, "taking into account the severity and very low prevalence of SOD1-ALS and the significant unmet need for effective treatments for this life-threatening disease." The Committee will also discuss whether clinical data from applicant Biogen’s placebo-controlled study and available long-term extension study results, with additional supporting results from the effects on relevant biomarkers, provide "convincing evidence of the effectiveness of tofersen in the treatment of patients with SOD1-ALS," the document states. Reference Link