SOD1-ALS therapy Qalsody moves closer to public funding in Canada
Biogen's gene-targeted treatment received conditional approval in March
- Qalsody, a gene-targeted therapy for SOD1-ALS, is nearing public funding in Canada.
- It reduces toxic SOD1 protein, which is expected to slow ALS progression.
- Reimbursement is contingent on a price reduction.
Qalsody (tofersen) is one step closer to being covered by public Canadian healthcare systems when used to treat adults with amyotrophic lateral sclerosis (ALS) who carry mutations in the SOD1 gene (SOD1-ALS).
Developed by Biogen, the therapy was conditionally approved by Health Canada in March, allowing it to be marketed based on positive preliminary clinical trial data. Its full approval is now contingent on evidence confirming that Qalsody can indeed improve outcomes for people with SOD1-ALS.
The move toward public reimbursement of Qalsody in Canada follows 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) in Québec.
“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,” Eric Tse, general manager of Biogen Canada, said in a company press release. “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.”
Qalsody has similar conditional approval in the U.S. and is approved in the European Union under exceptional circumstances.
Qalsody reduces production of toxic protein
ALS is a disease characterized by the progressive degeneration of motor neurons, the nerve cells in the brain and spinal cord that control muscle movement. In some cases, the disease is linked to mutations in the SOD1 gene, which result in the production of a faulty SOD1 protein that is prone to forming toxic aggregates in motor neurons, ultimately causing their death.
Administered via monthly injections directly into the spinal canal, Qalsody is designed to break down the genetic template required for the production of the SOD1 protein. By reducing production of this toxic protein, the therapy is expected to slow disease progression in people with SOD1-ALS.
Data from the Phase 3 part of the VALOR clinical trial (NCT02623699) and its open-label extension study (NCT03070119) largely supported Qalsody’s approval in Canada and elsewhere.
While initial VALOR results did not show a significant slowing in ALS progression in treated adults, data from the full trial and its extension phase revealed that starting Qalsody earlier was associated with a slower disease progression and a reduced risk of death. Treatment also correlated with slower declines in lung function and muscle strength.
In other assessments, Qalsody treatment was associated with reductions in neurofilament light chain (NfL) levels, an established biomarker of nerve damage. Patients who experienced early decreases in this blood biomarker tended to have slower disease progression.
Reimbursement will depend on price reduction
According to the recommendation from CDA, Qalsody should be reimbursed only when prescribed by an ALS specialist, such as a neurologist or physiatrist, within a multidisciplinary clinic.
The reimbursement also depends on a reduction in the price of Qalsody, which is currently expected to cost about CA$425,560 ($308,609 U.S.) per patient in the first year of treatment and CA$368,819 ($267,462) per patient in subsequent years.
The CDA said its health economic evidence showed that Qalsody did not represent good value for the healthcare system at the public list price. Even so, the recommendation also cited the urgent need for new treatment options for this rare form of ALS.
INESSS also acknowledged Qalsody’s clinical value, but the agency did not recommend listing the therapy at this time due to economic factors. Nevertheless, the review noted that clinicians consulted by INESSS considered some VALOR findings clinically meaningful, especially given the challenges faced by people with SOD1-ALS and the limited effective treatment options.
The arrival of Qalsody signals continued progress in ALS research and responds to the ongoing need for new treatment approaches.
As a result, the agency expressed its readiness to recognize Qalsody pending an agreement with the Québec Minister of Health.
“The HTA assessment process provides an important opportunity for the ALS community to have their voices heard,” said Tammy Moore, CEO 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.”
Biogen will now enter negotiations with the pan-Canadian Pharmaceutical Alliance to establish national pricing and access terms. If an agreement is reached, each province and territory will then decide individually whether to fund the medication and add it to their public reimbursement lists.
“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,” Moore said.
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