Alberta provides treatment for genetic disorder

By Dale Woodard on January 30, 2021.

Alberta families of children with a rare genetic disorder received some good news on the treatment front Wednesday when it was announced children suffering from spinal muscular atrophy may now be eligible to receive funding for gene replacement therapy treatment.
Alberta’s government is working with Novartis Pharmaceuticals Canada Inc. to provide interim patient access to Zolgensma.
Spinal muscular atrophy is a rare and progressive genetic disorder that causes muscle wasting. Alberta children with SMA and who may become ineligible for the treatment while waiting for the final approval processes to be completed will be considered for funding on a case-by-case basis.
“(It) will have a real impact on a small number of families in Alberta who have been waiting for good news related to their children’s health,” said Alberta Minister of Health Tyler Shandro in the announcement Wednesday morning. “In the last several months the stories of Alberta children who are impacted by spinal muscular atrophy have been shared in the news and over social media.”
Spinal muscular atrophy is a rare motor neuron disorder that can affect the muscles used for head and neck control, sitting, crawling, walking and swallowing. Zolgensma is administered by intravenous infusion to replace a missing or faulty gene. A one-time treatment of Zolgensma costs US $2.125 million.
“Until recently, there have been limited options to treat SMA,” said Shandro. “Although effective, available treatments require hospital visits for kids to receive drugs through a spinal tap, which can be painful, and requires maintenance dosing several times a year.”
Zolgensma was approved by Health Canada in December 2020 and a review by the Canadian Agency for Drugs and Technologies in Health is underway.
“Today I’m thrilled to be able to share another step forward as the Alberta Government has worked with Novartis Pharmaceuticals Canada Inc. to find a way to support these families,” said Shandro. “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. Because there is a process we must follow, we will continue to work with our national health technology agency and other provinces through the Pan Canadian Pharmaceutical Alliance towards a long-term agreement for access to Zolgensma for other children and their families.”
Specialist physicians who provide care to children with spinal muscular atrophy are able to submit an application on behalf of their patients for coverage to be considered.
“This is a positive step forward in providing better care for young children with spinal muscular atrophy and wonderful news for those families who will have access to this gene therapy,” said Dr. Jean K. Mah, Division of Pediatric Neurology, Alberta Children’s Hospital.
Susi Vander Wyk, executive director of Cure SMA Canada noted time is crucial for families afflicted with SMA.
“These families are going from a mindset of fear of what the future holds for their children and knowing the clock is ticking to planning the future and making plans for the entire family. This disease does not affect just the child, it affects the families and parents are devastated.”
“Thank you to the Alberta government for making a life-saving decision that will change the course of this devastating disease. We are thrilled knowing the impact of this decision on the patients and their families.”

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