'We're ecstatic': Alberta family wins drug lottery for toddler's rare condition

Alberta toddler wins drug lottery for rare condition CTV News Edmonton’s Dan Grummett has more on Alberta toddler’s win in a drug lottery for a rare condition.

EDMONTON — An Alberta family is rejoicing after winning a random drug lottery, giving their toddler a one-time drug treatment for a rare condition.

The Hanki family received the call on Wednesday informing them that their 15-month-old Harper was selected for Zolgensma – a one-time gene replacement therapy proven to halt, and in some cases reverse, the effects of spinal muscular atrophy.

“We're ecstatic. I mean, this is never anything we ever expected,” said Harper's father, John Hanki. “It's a lottery, I don't know who ever expects to win a lottery.

“To get that call just changes everything for us.”

The drug is not approved for use in Canada and costs $2.8 million south of the border.

Zolgensma’s manufacturer, Novartis, says in the U.S., the drug is "priced according to the value it provides to the patients, caregivers, healthcare system and to society as a whole."

John says an antibody test will be done in the next week, with Harper’s dose of the drug taking place at the end of November or early December in Calgary.

“We hope that she’ll walk and we understand she’ll likely won’t walk in a mall but walk from her bed to her closet or transfer herself to use the washroom,” added her mother, Amanda Hanki.

“It will change the course not only for our family, but for her as well. We are now living on borrowed time and I cannot wait to see what she does with it.”

The win for the Hankis is welcomednews to other families in the province with children battling the condition.

Reign, an 18-month-old boy whose condition now requires round-the-clock breathing assistance and a feeding tube, did not qualify for the lottery.

"It’s so awesome seeing all these Canadian babies getting the Zolgensmathrough the lottery, it’s so awesome that there is this lottery to provide the drug for free,” said Alex Johnston, Reign’s mother.

“But it is really, really disappointing and it has been since the beginning just to know that Reign doesn’t even get a chance at it.”

The only treatment currently allowed in Canada is a drug named Spinraza, which requires three yearly spinal injections for a patient’s life.

Alex tried to get Reign into the special access program last month, but was denied as well, due to him being on another medication.

The family says they are now forced to seek treatment outside of Canada, and have set up a GoFundMe, raising more than $100,000.

“The last grasp of hope was to get him into the special access program just in case we could get it administered here. But there’s nothing else we can do at this point. Fundraising really is our last option,” said Alex.

With files from CTV News Edmonton’s Dan Grummett and CTV News Edmonton Digital Journalist Alex Antoneshyn