Ionis says FDA advisors vote in favor of potential tofersen accelerated approval

Ionis (IONS) announced the outcome of the U.S. Food and Drug Administration’s Peripheral and Central Nervous System Drugs Advisory Committee meeting on tofersen, an investigational antisense medicine for the treatment of people with superoxide dismutase 1, or SOD1, amyotrophic lateral sclerosis, or ALS. On the question, "Is the available evidence sufficient to conclude that a reduction in plasma neurofilament light chain 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, 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. "We are encouraged by the outcome of today’s advisory committee meeting. If approved, tofersen would be the first medicine targeting a known cause of familial ALS, a devastating neurodegenerative disease that diminishes motor function and leads to death within two to five years of diagnosis," said C. Frank Bennett, Ph.D., executive vice president and chief scientific officer at Ionis. The company added: "Advisory Committees provide non-binding recommendations for consideration by the FDA. The New Drug Application for tofersen for the treatment of SOD1-ALS was submitted to the FDA for consideration under accelerated approval. The FDA is continuing its review of tofersen with a Prescription Drug User Fee Act, or PDUFA, action date of April 25, 2023."