Cystic fibrosis drug now offered to kids 6 and up in New Brunswick

·3 min read
Trikafta is used to correct a gene mutation. (Cystic Fibrosis Canada - image credit)
Trikafta is used to correct a gene mutation. (Cystic Fibrosis Canada - image credit)

New Brunswick children aged six and up who have cystic fibrosis are now eligible for a "transformational" drug.

Trikafta could help about 90 per cent of cystic fibrosis patients by correcting a cystic fibrosis genetic mutation called F508del, said Kim Steele, director of government and community relations for Cystic Fibrosis Canada.

"It is the single greatest innovation in the history of the disease," she said. "I mean, finding the gene was a significant scientific enabler to us being able to treat the defects in the gene."

Jodi O'Neill of Bath, the father of 16-year-old cystic fibrosis patient Colton, said the drug helped almost immediately after his son started it in March.

"He came down the stairs the next morning after starting it, and he said he could breathe easier. He noticed a difference straight away, just overnight."

Cystic fibrosis is a genetic disease that causes mucus to build up in the lungs, digestive tract and other parts of the body. This can make the body more susceptible to lung infections, and a double lung transplant is often required, said Steele.

O'Neill said the expansion of Trikafta eligibility to kids aged six to 11 will create a better quality of life for cystic fibrosis patients.

But although the drug does amazing things, it can't reverse damage that is already done," she said. She said if someone spends years in the hospital for repeat infections, there's going to be permanent damage.

"This is why we have newborn screening for cystic fibrosis, so we can start treatment as soon as possible before that damage begins," said Steele.

"That is what we are seeing our government do now is recognize that this drug is pivotal to an individual's future wellness and overall well-being."

New Brunswick approach

When Trikafta was introduced, it was approved for those 12 and older. In April, Health Canada approved the drug for children six or older, but it was up to individual jurisdictions to take the next step.

Even when the drug was limited to those 12 and older, New Brunswick was one of four provinces that did not require eligible patients to meet a starting lung function criterion to be able to access the drug, according Cystic Fibrosis Canada.

New Brunswick's expanded access to a larger age group became effective July 28.

CBC asked the Department of Health about the expanded program but got no response.

Opening up the future

Steele said New Brunswick was "rather quick" in its decision to expand the eligibility to include younger childen.

"There are still jurisdictions that we're waiting on to hear from," she said. "What is great is that people who need this drug for six years of age and older in New Brunswick, they can now work with their clinicians to get it."

Steele said by extending the drug to children, more "remarkable" health outcomes open up. More people with the gene can now see themselves having babies, and more people have come off the lung transplant list, she said.

"With the kids, we're really hoping that this drug will delay some of the significant health challenges that most people with cystic fibrosis have just living life every day."