Alex Johnston's 22-month-old son Reign was born with Type 1 SMA and currently requires 24-hour care.family handout/Courtesy of the family
The Alberta government has approved funding to treat a rare and often fatal disease in young children with a gene therapy that is considered the world’s most expensive pharmaceutical drug.
Health Minister Tyler Shandro said the province would pay for Zolgensma, which treats Type 1 spinal muscular atrophy, or SMA, on a case-by-case basis while provincial governments negotiate with the drug maker to work out long-term funding. A one-time treatment of Zolgensma costs about US$2.1-million. Alberta is the second province after Ontario to announce such interim funding.
Mr. Shandro said the government wants to ensure that young patients don’t lose eligibility while they wait for a final decision on funding. U.S. Food and Drug Administration (FDA) regulations don’t allow Zolgensma to be administered for children over two years of age, with a similar timeline expected in Canada.
“We’re focusing on kids not falling through the cracks,” he said. “The case-by-case basis is making sure that nobody is coming up to an age that would then adversely affect their eligibility criteria after there is funding.”
SMA is a rare neurological disease that affects one out of 11,000 babies, often leading to devastatingly rapid muscle deterioration and loss of breathing function. Without treatment, babies diagnosed with Type 1 SMA have a life expectancy of around two years.
Zolgensma is a one-time infusion that replaces the missing or defective survival motor neuron 1 (SMN1) gene, potentially reversing the progression of the disease.
The drug is not a cure, but in two clinical trials, it gave more than half of the 36 patients the strength to sit on their own. Those trials prompted the FDA to approve Zolgensma in 2019.
Before last month, the only drug for SMA approved by Health Canada was Spinraza, which costs $708,000 in the first year and $354,000 in each subsequent year – and must be taken for life.
Health Canada approved Zolgensma in December, leaving the issue of funding in the hands of the provinces. The Common Drug Review board of the Canadian Agency for Drugs and Technologies in Health (CADTH) is now reviewing Zolgensma and is expected to make recommendations to the provincial governments about funding later this year.
That slow process has meant an excruciating wait for families whose children have Type 1 SMA. Some have turned to fundraising through online sites such as GoFundMe to cover the drug’s high price tag.
Alex Johnston, whose 22-month-old son Reign was born with Type 1 SMA and currently requires 24-hour care, said she was elated by the news Alberta would fund Zolgensma on an interim basis. She and her fiancé, Ryan Fengstad, were worried they wouldn’t be able to get funding before Reign’s second birthday – only six weeks away – but are now hopeful they may have access to the drug before then.
“It’ll definitely increase his quality of life,” she said. “We can see him coming off the BiPAP [machine]. We can see him rolling and crawling and then hopefully eventually walking, eating on his own – all of those things.”
The family has been crowdfunding for support, but the $140,000 raised thus far is still nowhere near what it would take to pay for Zolgensma on their own.
The Swiss pharmaceutical company that makes Zolgensma, Novartis, launched a lottery a year ago to give away as many as 100 doses of the treatment in countries where the drug is not yet approved, including Canada.
Novartis spokeswoman Julie Schneiderman said the company is working with other provinces on the issue of interim access during negotiations over long-term funding. She said she couldn’t say whether provincial governments in Canada will end up paying the same US$2.1-million price that is charged in the United States, or what the company is charging Alberta and Ontario for the drug in the meantime.
Susi Vander Wyk, executive director of Cure SMA Canada, said the funding announcement comes as a relief for those caring for children with the disease.
“These families are going from a mindset of fear of what the future holds for them and knowing that this clock is ticking, to one of planning for the future and making plans for the entire family,” she said.
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