Canadian HTA Assessments Recognize Therapeutic Value of QALSODY™ (tofersen injection) and its Potential to Modify Disease Progression in Individuals Living with SOD1-ALS

• CDA issues positive reimbursement recommendation for QALSODY
• INESSS recognizes QALSODY's promising clinical value and highlights path toward reimbursement
• HTA assessments highlight importance of timely and equitable access to QALSODY for this ultra-rare genetic form of ALS and urgent need for therapies that slow progression, preserve function and independence, extend survival, and improve quality of life

TORONTO, Dec. 11, 2025 /CNW/ - Biogen Canada Inc. is encouraged by the recent Health Technology Assessment (HTA) evaluations issued by Canada's Drug Agency (CDA) and the Institut national d'excellence en santé et en services sociaux (INESSS) for QALSODY™ (tofersen injection), the first therapy designed to target a known genetic cause of amyotrophic lateral sclerosis (ALS).

CDA Issues Positive Reimbursement Recommendation¹
CDA, through the Canadian Drug Expert Committee (CDEC), issued a positive reimbursement recommendation for QALSODY for adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene. The recommendation highlights CDEC evaluations that data from the Phase 3 VALOR trial and its open-label extension suggest a clinical benefit with earlier initiation of QALSODY (compared to delayed treatment) in slowing functional decline and improving respiratory outcomes. The recommendation also cites the urgent need for new treatment options for this ultra-rare form of ALS.

INESSS Highlights Therapeutic Value and Path Toward Reimbursement²
INESSS acknowledged QALSODY's clinical value referencing several promising observations, including exploratory indications of slower decline in pulmonary function and reductions in neurofilament light chain, a biomarker of neuronal injury, but did not recommend listing QALSODY at this time due to economic factors. The review highlights that the clinicians consulted by INESSS described some of the findings as clinically meaningful given the substantial challenges faced by individuals living with SOD1-ALS and limited effective treatment options. As a result of its findings, the agency also expressed its readiness to recognize QALSODY for SOD1-ALS and to implement clinical follow-up and reassessment, pending an agreement with the Quebec Minister of Health.

Together, these assessments recognize the therapeutic value of QALSODY in treating SOD1-ALS and reinforce the importance of timely access for Canadians living with this ultra-rare disease.

"The HTA assessment process provides an important opportunity for the ALS community to have their voices heard," said Tammy Moore, Chief Executive Officer of the ALS Society of Canada. "The arrival of QALSODY signals continued progress in ALS research and responds to the ongoing need for new treatment approaches. The evaluation by CDA and INESSS highlight how critical timely and fair access to new therapies will be for families facing this devastating disease. We urge provinces to do their part by recognizing the value of innovative treatments like QALSODY and taking steps to ensure Canadians with SOD1-ALS are not left without options."

"We value the thorough assessments conducted by both CDA and INESSS, which acknowledge QALSODY's therapeutic value and the potential benefits it can bring individuals living with this devastating disease," said Eric Tse, General Manager of Biogen Canada. "Biogen remains dedicated to collaborating with partners and payors across Canada to support timely and equitable access to QALSODY and to offering people living with SOD1-ALS the hope of delaying disease progression and maintaining their functional independence and quality of life."

About QALSODY™ (tofersen injection) in Canada
QALSODY™ (tofersen injection) is an antisense oligonucleotide (ASO) designed to bind to SOD1 mRNA to reduce SOD1 protein production.

QALSODY is the first therapy designed to address the underlying genetic cause of SOD1-ALS³, a rapidly progressive and uniformly fatal condition that impacts motor function, speech, and respiratory ability.⁴ The conditional approval acknowledges plasma neurofilament light chain (NfL) as a marker of neurodegeneration⁵ 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.

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).⁴  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 asymptomatic SOD1-ALS variant carriers will serve as the confirmatory trials. More details about ATLAS (NCT04856982) can be found at clinicaltrials.gov.

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. Canada's Drug Agency. Reimbursement Recommendation (Final): Tofersen (Qalsody). Available at: https://www.cda-amc.ca/sites/default/files/DRR/2025/SR0883-Qalsody-Recommendation.pdf
   
   
2. Institut national d'excellence en santé et en services sociaux (INESSS). Advice to Minister about QALSODY. Available at: https://www.inesss.qc.ca/en/themes/medicaments/drug-products-undergoing-evaluation-and-evaluated/extract-notice-to-the-minister/qalsody-sla-avec-mutation-gene-sod1-7475.html
   
   
3. QALSODY™ Canadian Product Monograph. biogen.ca/products/QALSODY_PM_EN
   
   
4. 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.
   
   
5. Yuan A, Rao MV, Veeranna, Nixon RA. Neurofilaments and Neurofilament Proteins in Health and Disease. Cold Spring Harb Perspect Biol. 2017;9(4):a018309.

SOURCE Biogen Canada Inc.

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