QALSODY: A New Hope for ALS Faces a Hard Economic Reality

TORONTO, ON – December 11, 2025

For the first time, a therapy designed to target the underlying genetic cause of a rare and devastating form of amyotrophic lateral sclerosis (ALS) has arrived in Canada, offering a glimmer of hope where there was once only a grim prognosis. Biogen’s QALSODY™ (tofersen), a treatment for the 2% of ALS patients with a mutation in the superoxide dismutase 1 (SOD1) gene, represents a monumental leap in neurological science. Yet, this medical breakthrough has immediately collided with the fiscal realities of public healthcare, creating a complex and uncertain path for the very patients it was designed to save.

Following its conditional approval by Health Canada, QALSODY faced two critical evaluations by the nation's health technology assessment (HTA) bodies, and their divergent conclusions have exposed the deep-seated tension between embracing innovation and managing public expenditure. The result is a fractured landscape where a patient's access to a life-altering drug may depend on their postal code and the price their province is willing to pay for hope.

A Landmark Therapy for a Relentless Disease

ALS is a universally fatal neurodegenerative disease that relentlessly strips individuals of their ability to move, speak, and ultimately, breathe. While most cases are sporadic, a small fraction are genetic. SOD1-ALS is one such form, an ultra-rare diagnosis that can be particularly aggressive. Certain mutations, like the common A4V variant, can lead to a life expectancy of less than two years from symptom onset.

Until now, treatments have only modestly slowed progression. QALSODY operates on a different plane. As an antisense oligonucleotide, it is designed to reduce the production of the toxic SOD1 protein, tackling the root cause of the disease. Its approval was based on the Phase 3 VALOR trial and its open-label extension, which showed promising results. While the primary endpoint of functional decline at 28 weeks wasn't met with statistical significance, the therapy demonstrated a notable reduction in neurofilament light chain (NfL), a key biomarker of nerve damage, suggesting it was slowing the disease process. This evidence was compelling enough for Health Canada to grant conditional approval, pending results from ongoing confirmatory trials like the ATLAS study.

For families grappling with a SOD1-ALS diagnosis, the therapy's arrival signals a paradigm shift. "The arrival of QALSODY signals continued progress in ALS research and responds to the ongoing need for new treatment approaches," said Tammy Moore, CEO of the ALS Society of Canada. Her words underscore a sentiment felt across a community desperate for meaningful interventions.

A Fractured Verdict from Canada's Gatekeepers

The initial optimism following regulatory approval was quickly tempered by the HTA process. Canada's Drug Agency (CDA), which provides recommendations to all provinces except Quebec, issued a positive reimbursement recommendation. However, this green light came with significant conditions. The CDA stipulated that QALSODY should only be covered for adults with a confirmed SOD1 mutation and clinical symptoms, and, crucially, that its manufacturer, Biogen, must agree to a substantial price reduction. The agency’s analysis concluded that the drug was not cost-effective at its submitted price, even when considering the high unmet need.

In stark contrast, Quebec’s provincial agency, the Institut national d'excellence en santé et en services sociaux (INESSS), did not recommend listing the drug for public reimbursement at this time. While acknowledging QALSODY's "promising clinical value" and the meaningful benefits observed by consulting clinicians, INESSS balked at the price tag. The agency’s economic analysis was stark: covering the drug for an estimated eight patients in Quebec would cost the healthcare system approximately $5.3 million over three years. Citing an unfavorable ratio between cost and efficacy, INESSS put the brakes on access, even as it left the door open for future negotiations with the health minister.

This split decision creates a potential two-tiered system of access in Canada, highlighting a systemic challenge in evaluating drugs for ultra-rare diseases. While one agency deemed the clinical promise sufficient to proceed with price negotiations, the other found the economic burden too high to justify, leaving patients and their families in a painful limbo.

The Billion-Dollar Question: What is the Price of Progress?

The case of QALSODY is a microcosm of a global dilemma: how to sustainably fund breakthrough therapies for small patient populations. The research and development costs for such drugs are immense, and manufacturers argue that high prices are necessary to recoup investment and fund future innovation. In the United States, Biogen priced QALSODY at an annual cost of approximately $185,000 after an initial loading-dose period. This figure, while high, is in line with other recently launched ALS treatments.

However, publicly funded systems like Canada's operate on cost-effectiveness thresholds that drugs for rare diseases almost never meet. The standard willingness-to-pay threshold of around $50,000 per quality-adjusted life year (QALY) is dwarfed by the projected costs of these therapies. This forces HTA bodies and provincial payers into difficult, value-laden judgments.

"The evaluation by CDA and INESSS highlight how critical timely and fair access to new therapies will be for families facing this devastating disease," Moore noted, urging provinces to recognize the drug's value. Biogen, for its part, has experience navigating these waters with other high-cost therapies like Spinraza for spinal muscular atrophy and offers patient support programs like Biogen One™ to help with financial and logistical hurdles. "Biogen remains dedicated to collaborating with partners and payors across Canada to support timely and equitable access to QALSODY," stated Eric Tse, General Manager of Biogen Canada.

Yet, this collaboration is now entering its most critical phase. The positive CDA recommendation triggers negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) to establish a national price. The outcome of these confidential talks, and Quebec's subsequent decision, will determine whether the scientific achievement of QALSODY translates into tangible benefits for Canadian patients. This single negotiation will not only decide the fate of QALSODY's accessibility but will also set a powerful precedent for how Canada confronts the next wave of high-cost, high-impact genetic medicines that are just over the horizon.