Canada drug agency backs expanding coverage for SMA treatment
Draft guidance on Spinraza backs reimbursing adults with types 2 and 3
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- Canada's Drug Agency recommended expanding public funding for Spinraza to include adults and more patients with SMA.
- The draft guidance suggests covering adults with SMA types 2 or 3 and patients with two, three, or four SMN2 gene copies.
- Final reimbursement decisions depend on individual provincial governments after the review concludes.
Canada’s Drug Agency (CDA) recommends expanding public funding for Biogen’s Spinraza (nusinersen) to include adults and more patients with spinal muscular atrophy (SMA).
Provincial governments have yet to decide on funding.
Still in draft form, the recommendation is to include adults with SMA type 2 or type 3 and patients with two, three, or four copies of the SMN2 gene, which acts as a backup to SMN1, the gene that is mutated in SMA. Public feedback was collected until the end of last week, and a final recommendation is expected on Dec. 1.
“The CDA’s draft recommendation is a significant turning point for many in the Canadian SMA community,” Eric Tse, general manager of Biogen Canada, said in a company press release. “Adults with SMA and individuals with four copies of SMN2 have aspired to access treatment, and we’re thrilled to welcome this milestone.”
‘Years of hope and disappointment’
In SMA, mutations in SMN1 result in little or no SMN protein being produced. SMN is needed for the survival of motor neurons, the nerve cells that control muscles. As motor neurons become damaged, muscle weakness worsens over time. Typically, more severe symptoms usually occur in patients with fewer copies of SMN2, which produces a smaller amount of functional SMN protein than healthy SMN1.
Spinraza contains a short strand of genetic material, called an antisense oligonucleotide, which is designed to correct how SMN2 is processed, allowing the body to produce more functional SMN protein. Although Spinraza is approved to treat all types of SMA, reimbursement policy in Canada does not include adults or patients with four copies of the SMN2 gene.
CDA’s draft recommendation supports public funding for Spinraza for patients with genetically confirmed SMA who have two, three, or four copies of the SMN2 gene, including adults, provided certain conditions are met. Although Spinraza has been approved in Canada since 2017, only Quebec has covered treatment for all ages and types of SMA since 2018.
One patient living with SMA type 2 described “years of hope and disappointment as SMA treatments became approved for us in Canada but remained publicly inaccessible due to restrictive eligibility criteria and provincial funding decisions. He finally gained access to treatment after extensive advocacy, media attention, and political intervention,” according to the draft recommendation.
Recommendation follows thorough review
The recommendation follows a review that began in March through a pilot project by the CDA’s Formulary Management Expert Committee (FMEC). The committee read clinical studies, real-world data, medical registries, and feedback from patients, families, and specialists to conclude whether existing funding rules should change.
The evidence suggests that Spinraza may stabilize or improve muscle function in adults and patients with four or more copies of the SMN2 gene. Although the evidence remains limited, the FMEC considered the progressive nature of SMA and the difficulty of conducting large, randomized controlled studies in making its recommendation.
The draft recommendation is a landmark moment for the SMA community and a testament to the incredible determination of patients and families who have tirelessly worked to ensure their voices were heard.
To be eligible for reimbursement, patients should be prescribed Spinraza by a neurologist with expertise in SMA. It should be used alone as a maintenance treatment or temporarily while a patient transitions to another treatment, such as Zolgensma (onasemnogene abeparvovec-xioi), after which it should be stopped. Spinraza should also be discontinued if the disease progresses significantly or side effects become unacceptable. The FMEC also recommends a price reduction.
Patient organizations, including Cure SMA Canada, Muscular Dystrophy Canada, and the Canadian Organization for Rare Disorders, were in charge of gathering patients’ perspectives.
“The CDA’s draft recommendation follows years of advocacy by SMA community members who bravely stepped forward to share their experiences,” said Susi Vander Wyk, executive director of Cure SMA Canada. “More work is ahead, but right now we mark a truly meaningful advancement for the SMA community.”
For Stacey Lintern, CEO of Muscular Dystrophy Canada, “the draft recommendation is a landmark moment for the SMA community and a testament to the incredible determination of patients and families who have tirelessly worked to ensure their voices were heard.”
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