Alberta announces funding for pricey gene therapy drug Zolgensma

CALGARY — Alberta Health Minister Tyler Shandro announced funding Wednesday for the gene therapy drug Zolgensma, which is used to treat toddlers with spinal muscular atrophy (SMA).

The drug was approved by the federal government in December. Before that, patients could only access it through a federal special access program at a cost of roughly $2.8-million per year.

Funding from the province will be done on a case-by-case basis, said Shandro.

“There is no budget cap, what we’re looking for is focusing on kids not falling through the cracks as we’re waiting for the rest of the processes that need to be done for drugs to become approved and listed in the formulary here in Alberta,” he said.

“So the case-by-case basis is making sure that nobody is coming up to an age that would then adversly affect their eligibility criteria after there is funding.”

Several Alberta families have been fundraising to try and cover the costs of treatment for months. Other Canadian families with children who suffer from SMA entered into a lottery for treatment held by Novartis.

“I cried,” said Bryarly Parker, a Fairview, Alta., mom whose son, Max, was diagnosed with Type 2 SMA at 22 months old.

“I smiled and cried, it was just an instant relief for us and for the families in Alberta who have come before us and fought for this. It’s incredible; it is a huge win for us.”

The family’s doctor still has to apply for the funding on their behalf and they still have to be approved. Because of that, Parker said they will continue to fundraise.

“With everything we’ve been doing, there’s not just one answer and there’s not just one thing that’s going to make it completely OK and everything is going to be great,” she said.

“That’s why it’s like climbing a mountain, there’s so many steps to climb and so many steps to get through.”

About one-in-10,000 Canadian babies are born with a type of SMA each year, according to the National Organization for Rare Disorders. The genetic neuromuscular disease is caused by a defective or missing SMN1 gene, which results in a loss of muscle function. The young patients diagnosed with SMA often have trouble breathing, swallowing and crawling.

Zolgensma is designed to replace the missing or defective SMN1 gene by delivering a new working copy of it into a patient’s cells via intravenous infusion, says Novartis. The therapy is supposed to stop the disease’s progression and restore SMN protein production.

“The earlier you receive this treatment, the better the prognosis,” said Susi Vander Wyk, executive director of Cure SMA Canada.

“So, we have children who were identified through newborn screening … that you can’t even tell they have SMA post-treatment with Zolgensma, whereas, without treatment, they would more than likely have passed on before the age of two.

“Children that have been treated a little bit later, we know there is examples of them not being able to move their arms and legs, and suddenty they’re rolling off a couch and sitting on the floor and reaching for things. With this treatment we know it gets better and better with time. It has an astounding impact on those babies.”

Thirteen sites in Canada have been cleared to administer Zolgensma: Vancouver, Edmonton, Calgary, Saskatoon, Winnipeg, London, Hamilton, Toronto, Ottawa, Montreal, Quebec City and Halifax.

With files from CTV Calgary’s Jordan Kanygin

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