Cystic fibrosis life-altering drug nearing patient access across the country: Health Canada

Click to play video: 'Cystic fibrosis drugs given green light for sale in Canada, one step closer to patient access'
Cystic fibrosis drugs given green light for sale in Canada, one step closer to patient access
WATCH: Health Canada approved the marketing of the drug called Trikafta across the country on Friday. Medical experts feel cystic fibrosis patients could have access to this life-altering drug by the end of 2021 – Jun 20, 2021

For the cystic fibrosis (CF) community, persistence and constant belief are paying off.

Health Canada announced on Friday that the cystic fibrosis drug Trikafta is now available to be prescribed by doctors across Canada.

At this point, the drug would be available to those aged 12 and over and patients who have at minimum one of the CF F508del gene mutations.

Cystic fibrosis affects 4,344 Canadians, according to the CF website, with 130 of those currently residing in Saskatchewan.

Every newborn in Canada is tested most commonly by a sweat test.

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Health Canada says for those under the age of 12, progression and research continue for the approval of the younger CF patients.

According to the cystic fibrosis website, Trikafta could over the span of time reduce the number of people living with severe lung disease by 60 per cent and the number of deaths by 15 per cent. It would also add years of life to those with CF. For example, a child born with the disease would see a decade added to their lives.

CF affects mainly the respiratory system, particularly the lungs. As a way to counteract the inflammation that occurs, patients usually have a daily routine that involves devices that help get rid of the mucus build-up.

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In addition to Trikafta, the first and second-generation drugs Orkambi and Kalydeco were given the green light by the pan-Canadian Pharmaceutical Alliance (pCPA). The pCPA is responsible for negotiating prices on behalf of all the provinces. It was announced the negotiating price for the two CF drugs was agreed upon.

All three of these drugs are produced by American-based company Vertex.

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“The approval of TRIKAFTA marks a significant milestone for Canadians with CF, their families and Vertex,” Vertex Chief Executive Officer and President Reshma Kewalramani said in a statement.

CF patient and Saskatoon resident Emily Brennan took time to ponder the possibilities this drug could provide her, such as allowing for her to be a part of family life.

“I might actually get the chance to meet my grandchildren. I might get to be there with my husband as we grow old. I might get to see my son’s graduation (or even) his first date,” she said.

Brennan says she has had to do her CF medical treatments and therapies for up to eight hours per day while dealing with a recent lung infection. Last year, she spent a lot of time in the hospital or on home IV antibiotic therapies or related measures for roughly half the year.

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The Nechvatal family is no stanger to CF. Their two boys, Dominic (12) and Benjamin (5), have the rare and fatal genetic disease. Their father Shaun says this is the news those affected by CF have been waiting for since negotiations for these drugs began in 2012.

Click to play video: 'Saskatoon family waiting on new deal for access to cystic fibrosis treatments'
Saskatoon family waiting on new deal for access to cystic fibrosis treatments

The boys put in a handful of hours each day to clear their lungs of mucus with devices and dozens of pills and vitamins. Shaun says the drug corrects the protein to the point where a person with CF who’s given a baseline test would register negative for the disease.

“They will have the opportunity to live fuller lives then say if they required a lung transplant or something,” said Nechvatal. “With today being Father’s Day and being a father of two boys directly impacted by this news, (it’s the) best gift ever.

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CF advocate and founder of the Canadian Cystic Fibrosis Treatment Society Chris MacLeod says this is phenomenal news.

“It’s game-changing news. (Trikafta) is truly a life-saving drug. Life-changing,” he said.

MacLeod was granted exceptional drug status in 2012 after his lung function dropped to 30 per cent. He says if it wasn’t for the first-generation gene modulator Kalydeco, he more than likely would have died eight years ago. Instead, the drug doubled his lung function.

MacLeod added the only phase remaining in the negotiations is for each province to list each drug, which would allow for people within each province to have full access to them.

“If we don’t have CF patients on these drugs by Labour Day weekend, that would be shocking and a failure and we can’t do that.” said MacLeod.

Cystic fibrosis chief scientific officer Dr. John Wallenburg says these drugs would help out with roughly 90 per cent of those with CF. The remaining five to 10 per cent, or the rarer cases with unique CF gene mutations, would have to find alternative means of help such as cellular therapies. But, these have not been approved yet.

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He gives credit to the unrelenting push and drive from the CF community to get to this point. There are dozens of other countries that have approved Trikafta or even all three of the drugs.

“I think it’s going to be incumbent on us to inform the federal government and members of Parliament of the provincial legislatures of the importance of this decision,” Wallenburg said.

Medical director for Toronto adult CF Centre and U of T professor of medicine Dr. Elizabeth Tullis says the approach to tackling patients with CF is significant, as the drugs correct the broken proteins.

“For the first time with CFTR modulators, we are fixing the problem at the source. We are preventing the problem from developing, which is a huge change,” Tullis said over a Zoom call.

“If you have a huge hole in your ceiling and it’s dripping water, you can either mop up the water or fix the hole in the ceiling. Trikafta and all these other drugs are fixing the hole in the ceiling.”


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