Biogen Canada Inc. announced that Health Canada has accepted for review a New Drug Submission(NDS) for tofersen for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. If approved, tofersen will be the first treatment indicated in Canada to target a genetic cause of ALS.
"The tofersen New Drug Submission represents a critical milestone for the Canadian ALS community and those suffering from this ultra-rare genetic form of the disease that robs them of time with loved ones," said Eric Tse, General Manager of Biogen Canada. "For over a decade, Biogen has been committed to advancing ALS research to provide a deeper understanding of this devastating disease. We are passionate about doing what we can to make a difference and are deeply committed to the rare disease community."
According to the ALS Society of Canada, ALS currently impacts an estimated 4,000 Canadians, with approximately 1,000 new Canadians diagnosed each year.2
The NDS is based on results from the Phase 3 VALOR trial, a 28-week randomized, double-blind, placebo-controlled clinical study, which assessed the safety and efficacy of tofersen in patients with weakness attributable to ALS and a confirmed SOD1 mutation.
A regulatory decision on the New Drug Submission is expected in early 2025.
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