UPDATE: The Alberta government announced on Dec. 1, 2021, that it had reached a long-term pricing agreement with the manufacturer of Zolgensma, and the gene therapy treatment used for spinal muscular atrophy had been added to the Rare Diseases Drug Coverage Program.
The Alberta government said Wednesday it had reached an agreement with the manufacturer of Zolgensma, a drug used to treat the rare illness spinal muscular atrophy, or SMA, to fund the treatment for some children in the province.
Health Minister Tyler Shandro said this new agreement will allow the government to provide children and their families the drug, instead of waiting for the final steps in the Canadian approval process.
“I’m very aware of the urgency of making this treatment available and pleased that we are able to provide this funding for children who may slip through the cracks while waiting for the final review and final approval processes,” Shandro said.
Zolgensma, a gene therapy medication, was approved by Health Canada in December, however, Shandro said the standard drug review and negotiating processes are still not complete, which means families are still waiting to see how they can access it for their children.
Zolgensma is a one-dose medication that would replace the faulty gene at the root of the SMA disorder.
Shandro said Alberta is still working with the national health tech agency and the pan-Canadian Pharmaceutical Alliance to come up with a long-term plan to provide access to Zolgensma in Alberta.
For the time being, Shandro said specialists and physicians will be given information to help them and families determine which children are eligible to receive the treatment now and apply for it.
SMA is a disease that affects roughly one in 6,000 babies born, and attacks their muscular system, affecting their ability to develop and maintain motor skills like neck and head stability, sitting up, crawling, walking and even swallowing.
Shandro said the illness is often diagnosed when children are between three and six months old, and is detected when a baby doesn’t kick as vigorously as they should, or can’t bear weight on their legs without help.
“I can’t imagine the stress and hardship of families with a child diagnosed with SMA. As parents we want what’s best for our children,” Shandro said.
“We want them to have healthy development and have every opportunity available to them.”
For the family of Max Sych, also known as Mighty Max, it’s a glimmer of hope not just for them, but also for the other families they advocate for.
The family doesn’t yet know if Max will be eligible for the drug, but they’re looking into starting the approval process.
“It’s really, really exciting and it’s a huge win for the SMA families,” Max’s mom Bryarly Parker said Wednesday.
No matter what happens, she says they will keep advocating for treatment for the illness.
“It won’t end with Max. For me as a parent, what I’ve gone through at the NICU and now this — I won’t stop until there’s newborn screening. I won’t stop until every family that wants their child to have access gets this medication,” Parker said.
“We’re not going to stop fundraising, we’re not going to stop our advocacy until literally Max is in the hospital and I just see that Kangaroo pump push that IV into Max.”
Suzi Vander Wyk with Cure SMA Canada said this news will bring hope to families struggling with an SMA diagnosis, adding that the illness doesn’t just impact the child, but the entire family unit.
“These families are going from a mindset of fear of what the future holds for their children and knowing that this clock is ticking, to one of planning the future and making plans for the entire family,” she said.
Vander Wyk said because Zolgensma is a new treatment, it’s too early to know the long-term results for children who receive it, but said it does have “an astounding impact on those babies.”
She said if a child is diagnosed with SMA during newborn screening, before anyone can tell they have the illness, and they receive the treatment right away, there are no signs of SMA as they grow.
“Children that have been treated a little bit later, we know that there are examples of them not being able to move their arms and legs and suddenly they’re rolling off a couch and sitting on the floor and reaching for things, and with this treatment, we know that it gets better and better with time,” Vander Wyk said.