The Alberta Government has approved a treatment access option for Zolgensma, and says it will help families of children with spinal muscular atrophy.
The disease, and the Zolgensma treatment, has seen discussion around the province as people, including Lloydminster residents, rally around children like Max Sych, or Mighty Max, who is currently battling SMA.
On Wednesday, Health Minister Tyler Shandro announced the Alberta Government is working with Novartis Pharmaceuticals Canada, the manufacturer, to fund Zolgensma on a case-by-case basis for children who may become ineligible for the treatment while waiting for approval.
“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 the final approval processes.”
Spinal Muscular Atrophy type II is a rare neuromuscular disease in the part of the nervous system controlling voluntary muscle movement, people with SMA Type II will experience difficulty walking, sitting up on their own, swallow or breathe.
Zolgensma has been found to significantly increase length and quality of life for people with SMA Type II. However, families have faced challenges due to access and cost. The gene therapy must be performed before a child’s second birthday and was only approved by Health Canada in December 2020. It also costs $2.8 million.
Shandro says they’ll be working with the pan-Canadian Pharmaceutical Alliance to create a plan long-term agreement for access to the drug for other children and families.
Cure SMA Canada Executive Director Susi Vander Wyk is praising the decision in the meantime, saying it’s game-changing for children and families fighting SMA.
“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 ones of the future, making plans for the whole family, because it affects not only the child but the whole family as a unit.”
Zolgensma is an intravenous infusion to replace a missing or faulty gene and is one time only instead of ongoing treatment.