Health Canada Accepts for Review a NDS for Tofersen

Biogen Canada announces Health Canada accepts for review NDS for tofersen to treat rare, genetic form of ALS

Overview

Biogen Canada Inc., a leading biotechnology company, 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.

Words from Biogen

“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.”

ALS: Epidemiology

According to the ALS Society of Canada, ALS currently impacts an estimated 4,000 Canadians, with approximately 1,000 new Canadians diagnosed each year.

VALOR Trial

• 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.
• About Tofersen
• Tofersen is an antisense oligonucleotide (ASO) designed to bind to SOD1 mRNA to reduce SOD1 protein production. 
• The US Food and Drug Administration granted accelerated approval for Qalsody (tofersen) to treat amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with tofersen. 
• Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s). 
• Tofersen also received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommending its marketing authorization under exceptional circumstances for the treatment of adults with ALS associated with a mutation in the SOD1 gene. 
• A decision on a marketing authorization in the European Union is expected in the second quarter of 2024.

The license:- Biogen licensed tofersen from Ionis Pharmaceuticals, Inc. under a collaborative development and license agreement. Tofersen was discovered by Ionis.

About ATLAS Study

• In addition to an ongoing open label extension (OLE) of VALOR, tofersen is being studied in the phase 3, randomized, placebo-controlled ATLAS study to evaluate whether tofersen can delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity (elevated plasma NfL).

About Amyotrophic Lateral Sclerosis (ALS)

• Amyotrophic lateral sclerosis (ALS) is a rare, progressive, and fatal neurodegenerative disease that results in the loss of motor neurons in the brain and the spinal cord that are responsible for controlling voluntary muscle movement. 
• People with ALS experience muscle weakness and atrophy, causing them to lose independence as they steadily lose the ability to move, speak, eat, and eventually breathe. 
• Average life expectancy for people with ALS is three to five years from time of symptom onset.
• Multiple genes have been implicated in ALS. Genetic testing helps determine if a person’s ALS is associated with a genetic mutation, even in individuals without a known family history of the disease. 
• More than 15 per cent of people with ALS are thought to have a genetic form of the disease; however, they may not have a known family history of the disease.

About SOD1-ALS

• SOD1-ALS is diagnosed in approximately two percent of all ALS cases. 
• In people with SOD1-ALS, mutations in their SOD1 gene cause their bodies to create a toxic misfolded form of SOD1 protein. 
• This toxic protein causes motor neurons to degenerate, resulting in progressive muscle weakness, loss of function, and eventually, death.

About Biogen

Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patient lives and to create value for shareholders and our communities.

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