Woman with cystic fibrosis urges manufacturer to bring 'miracle drug' to Canada
A woman with cystic fibrosis is urging the manufacturer of a new “miracle drug” to make the medicine available in Canada.
Cystic fibrosis is a fatal genetic disease which mainly affects the lungs and digestive system. Its severity differs from person to person, but persistent infection in the lungs and the resulting loss of lung function, will eventually lead to death in the majority of people with CF, according to the non-profit Cystic Fibrosis Canada.
- Read more: 'This drug saved my life': Cystic Fibrosis patient accuses drug company, Canada of stalling on approval
Until recently, Chelsea Gagnon from West Island, Montreal, inhaled vapour to prevent infection by breaking up mucus in her lungs. But she has now started to take a drug called Symdeko, which she credits with saving her life.
"I'm certain that if I had I not started this medication, I would have deteriorated so much that I would have needed a double lung transplant or I would’ve been dead," Gagnon told CTV News Montreal.
"This medication saved my life. It changed my life and there's an even better version out right now and Canadians can't access any of them."
Symdeko stabilizes patient’s lung degeneration by fixing the protein deficiency at the root of the illness, but the drug is expensive, about $250,000 a year, and is not covered by Medicare. Luckily for Gagnon, it is included on her mother’s private insurance.
The better version Gagnon refers to is Trikafta, which can improve lung capacity.
“This drug is a medical breakthrough,” according to John Wallenburg, CF Canada’s chief scientific officer. “If you look at what happened in the United States for example, the FDA (Food and Drug Administration) chose to use four separate mechanisms to speed the approval of this drug and they succeeded in approving it in 90 days."
Trikafta manufacturer Vertex Pharmaceuticals has not submitted the drug for approval in Canada, a process that can take between two and five years.
"Every day that we delay, it’s a degenerative disease, so our lungs get worse and people do pass away, they wait for lung transplants, the transplants don't come,” Gagnon said.
“Life gets harder every day, breathing isn't easy. I'm going to be one of the last generations, when these modulators are available that are going to die from cystic fibrosis.
“I think the children who get the medication today are going to live pretty normal lives."
Gagnon told CTV News Montreal that she’ll use every last breath to make sure that happens sooner rather than later.
In a statement, Vertex said the company is unable to comment on drugs that haven’t received market authorization in Canada.
“Vertex has concerns that new Canadian medicine pricing rules, as they are currently written, have the potential to limit access to treatments for Canadians living with a rare disease,” it read.
“We are working closely with relevant stakeholders to re-envision an environment where access to rare disease medicines for all eligible patients is achievable.”
Health Canada and the Quebec Health Ministry did not respond to requests from CTV News Montreal for comment.
--- With files from CTV News Montreal’s Matt Gilmour