QALSODY™ (tofersen injection) Receives Conditional Marketing Authorization from Health Canada as the First ALS Treatment Targeting a Genetic Cause

• QALSODY targets the root cause of SOD1-ALS, an ultra-rare and uniformly fatal neurodegenerative disease marked by motor neuron loss in the brain and spinal cord¹
• Biogen's second rare disease therapy to be approved in Canada reflects a continued commitment to advancing innovative therapies for diseases with high unmet medical need

TORONTO, March 3, 2025 /CNW/ - Biogen Canada Inc. announced today that Health Canada has issued marketing authorization with conditions (Notice of Compliance with Conditions (NOC/c)) for QALSODY™ (tofersen injection) for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.  

QALSODY is the first therapy designed to address the underlying genetic cause of SOD1-ALS², a rapidly progressive and uniformly fatal condition that impacts motor function, speech, and respiratory ability.¹ This conditional approval acknowledges plasma neurofilament light chain (NfL) as a marker of neurodegeneration³ to support efficacy in SOD1-ALS and was granted based on the totality of evidence, including the targeted mechanism of action, biomarker and clinical data.

"This approval marks a significant advancement in our approach to ALS. QALSODY is the first therapy to directly target the root cause of SOD1-ALS, addressing the misfolded proteins responsible for the disease—a distinction that sets it apart from other ALS treatments, which primarily focus on symptom management rather than modifying the disease itself," said Dr. Angela Genge, a study investigator of the QALSODY clinical trials and director of the ALS Centre of Excellence for Research and Patient Care at McGill University. "Trial results demonstrate the potential for QALSODY to slow disease progression and show encouraging signs of improved function in some patients. These findings represent a significant step forward in managing this challenging disease for individuals with confirmed SOD1 mutations."

Authorization in Canada under the NOC/c guidance was granted based on the acceptable safety profile, high quality and promising efficacy of QALSODY observed in the 28-week randomized, double-blind, placebo-controlled Phase 3 VALOR trial, which enrolled 108 patients aged 23 to 78 years with weakness attributable to ALS and a confirmed SOD1 mutation, and its open-label extension (OLE).²  The authorization is conditional, pending the results of trials to verify its clinical benefit. The final OLE clinical report, summary of integrated analyses with VALOR trial, and results of the ongoing Phase 3 ATLAS study of tofersen in clinically presymptomatic SOD1-ALS patients will serve as the confirmatory trials.

"This announcement represents a pivotal moment for the ALS community and brings renewed hope to people living with SOD1-ALS," said Tammy Moore, Chief Executive Officer of ALS Society of Canada. "It highlights the ongoing progress in research, offering a new and promising approach to improving what it means to live with this life-altering diagnosis. We remain steadfast in our commitment to ensuring equitable access to innovative therapies, advancing the standard of care for all individuals living with this relentlessly progressive disease, and strive to champion the best possible outcomes for all patients."

SOD1-ALS is an ultra-rare, genetic form of ALS, comprising approximately 2% of people with ALS.⁴

"We're thrilled to bring forward QALSODY as a treatment for individuals living with SOD1-ALS, a rare form of this devastating disease," said Eric Tse, General Manager of Biogen Canada. "This approval not only marks a milestone in ALS research but also highlights our ongoing commitment to advancing therapeutic options for rare diseases and helping patient populations with high unmet medical need."  

About QALSODY™ (tofersen injection)
QALSODY™ (tofersen injection) is an antisense oligonucleotide (ASO) designed to bind to SOD1 mRNA to reduce SOD1 protein production. The U.S. Food and Drug Administration granted accelerated approval for QALSODY to treat amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).⁵ The European Commission granted marketing authorization under exceptional circumstances and orphan designation for QALSODY.⁶

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

QALSODY 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). More details about ATLAS (NCT04856982) can be found at clinicaltrials.gov.

About Amyotrophic Lateral Sclerosis and SOD1-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.⁷

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. Mutations in the SOD1 gene are responsible for approximately 2% of the estimated 168,000 people who have ALS globally (SOD1-ALS).⁴ More than 15 percent of people with ALS are thought to have a genetic form of the disease;⁸ however, they may not have a known family history of the disease.⁴

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.⁸

Biogen's Continuous Commitment to ALS
For over a decade, Biogen has been committed to advancing ALS research to provide a deeper understanding of all forms of the disease. The company has continued to invest in and pioneer research despite making the difficult decision to discontinue a late-stage ALS asset in 2013. Biogen has applied important learnings to its portfolio of assets for genetic and other forms of ALS, with the goal of increasing the probability of bringing a potential therapy to patients in need. These applied learnings include evaluating genetically validated targets in defined patient populations, pursuing the most appropriate modality for each target and employing sensitive clinical endpoints. In addition to tofersen, the company has a robust discovery pipeline including efforts to address TDP43 pathology for the broad ALS population. TDP43 pathology is seen in 97% of ALS cases and is considered a hallmark of the disease.

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. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment, to deliver long-term growth.

Biogen has been proudly serving Canadian patients for more than 25 years. For information about Biogen Canada, please visit www.biogen.ca. 

References:

1. Brown RH, Al-Chalabi A. N Engl J Med. 2017;377(2):162-172. 2. Masrori P, Van Damme P. Eur J Neurol. 2020;27(10):1918-1929. 3. Miller T, et al. N Engl J Med. 2020;383(2):109-119.
   
   
2. QALSODY™ Canadian Product Monograph. biogen.ca/products/QALSODY_PM_EN
   
   
3. Yuan A, Rao MV, Veeranna, Nixon RA. Neurofilaments and Neurofilament Proteins in Health and Disease. Cold Spring Harb Perspect Biol. 2017;9(4):a018309.
   
   
4. Brown CA, Lally C, Kupelian V, Flanders WD. Estimated Prevalence and Incidence of Amyotrophic Lateral Sclerosis and SOD1 and C9orf72 Genetic Variants. Neuroepidemiology. 2021;55(5):342-353. doi: 10.1159/000516752. Epub 2021 Jul 9.
   
   
5. QALSODY Prescribing Information, Cambridge, MA: Biogen. https://www.biogencdn.com/us/pdfs/qalsody-prescribing-information.pdf Accessed: January 2025.
   
   
6. European Medicines Agency, Qalsody Product Information. https://www.ema.europa.eu/en/documents/product-information/qalsody-epar-product-information_en.pdf  Accessed: January 2025. 2. European Medicines Agency, Orphan Designation for treatment of amyotrophic lateral sclerosis. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1732 Accessed: January 2025.
   
   
7. National Institute of Neurological Disorders and Stroke. Amyotrophic Lateral Sclerosis (ALS). Available at: https://www.ninds.nih.gov/health-information/disorders/amyotrophic-lateral-sclerosis-als.  Accessed: January 2025.
   
   
8. Akcimen F, Lopez ER, Landers JE, et al. Amyotrophic lateral sclerosis: translating genetic discoveries into therapies. Nat Rev Genet. 2023. https://doi.org/10.1038/s41576-023-00592-y

SOURCE Biogen Canada

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