QALSODY (tofersen)

Qalsody is available to eligible individuals through Health Canada’s Special Access Program (SAP).

On March 3, 2025, Health Canada approved QALSODY (tofersen injection) under a Notice of Compliance with Conditions (NOC/c) for the treatment of ALS in adults who have a pathogenic variant (also known as a mutation) in the superoxide dismutase 1 (SOD1) gene. This means the drug will soon be made available and can be marketed in Canada with certain conditions.

The NOC/c pathways allow for the authorization of treatments based on promising clinical data while requiring additional evidence to confirm the drug’s benefit. The authorization of QALSODY remains conditional and requires additional data from ongoing trials.

This approval is a positive step forward as QALSODY becomes the first therapy indicated in Canada to target a genetic cause of ALS. However, at this stage, the drug is not yet covered under public or private drug programs. As a next step, Canada’s Drug Agency (CDA) and Institut national d’excellence en santé et services sociaux (INESSS) in Quebec must complete its health technology assessment (HTA) of the therapy, informing whether it will be recommended for public reimbursement.

At this stage, Qalsody is not yet covered under public or private drug programs. As a next step, Canada’s Drug Agency (CDA) and Institut national d’excellence en santé et services sociaux (INESSS) in Quebec must complete its health technology assessment (HTA) of the therapy, informing whether it will be recommended for public reimbursement. Following this process, federal, provincial, and territorial drug plans, as well as private insurers, will determine if they will cover the costs associated with the drug.

ALS Canada remains actively engaged in the process. We are participating in CDA’s patient input process and working to support the pan-Canadian Pharmaceutical Alliance (pCPA) and the provincial drug programs to make informed and expedited reimbursement decisions by bringing forward the perspective and experiences of the ALS community. We are hopeful that pan-Canadian and provincial decision-makers in the reimbursement process will work quickly to provide expedited and equitable access for Canadians living with SOD1-ALS.

Qalsody (tofersen) is an investigational therapy for the treatment of ALS in adults with a pathogenic variant in the SOD1 gene.

The SOD1 gene encodes the information to make a protein called superoxide dismutase 1. This protein helps cells to function properly by clearing toxic byproducts called reactive oxygen species (ROS), also known as free radicals. These byproducts are created naturally in the cell after normal cellular processes and must be broken down regularly to prevent oxidative cell damage.

Many variants in this gene can lead to a defective version of this protein, which becomes toxic and disrupts normal cell biology, contributing to ALS. Variants in the SOD1 gene are believed to cause ten to thirty percent of familial ALS cases and approximately one to four percent of individuals with no obvious family history.

Tofersen is an antisense oligonucleotide (ASO) that targets SOD1 mRNA, leading to reduced production of the SOD1 protein and decreased toxic levels in cells.

In clinical trials, tofersen reduced SOD1 levels in the cerebrospinal fluid (CSF) of people living with SOD1-ALS and levels of NfL, a biomarker indicative of nerve cell damage.

Biogen’s ongoing ATLAS Phase 3 clinical trial aims to further evaluate the efficacy of the therapy and determine whether early treatment with tofersen can delay the onset of ALS symptoms and/or significantly slow decline in function after symptoms appear. Individuals with SOD1-ALS participating in the ATLAS study will be monitored regularly for biological changes that indicate the underlying disease processes have started. This is the first clinical trial to ever test a treatment in individuals who have not yet been diagnosed with ALS.