Cystic fibrosis: they desperately await approval of a drug

Sherbrooke residents, like other cystic fibrosis patients in Canada, are impatiently awaiting the approval by the authorities here of a drug used in the United States, Trikafta.

William Côté was just a few months old when he was diagnosed with cystic fibrosis. The Sherbrooke resident is now 23 years old and his lung capacity is 48%. For him, access to the drug Trikafta would change his life.

“Not only could it increase my lung capacity, but it would also extend my life expectancy,” he said. I could be with my wife and kids longer. ”

The drug Trikafta has been available in the United States since the fall of 2019. A dispute between the pharmaceutical company Vertex and Health Canada is believed to be the cause of the delay.

On the Cystic Fibrosis Canada website, we can read: “According to a study published in August 2020, early access to Trikafta would significantly improve the health of people with fibrocysts”.

“The study shows that if Trikafta were to become available in 2021, the estimated median survival age of a child born with CF in 2020 would increase by 9.2 years,” we also read. By 2030, Trikafta could reduce the number of people with CF with severe lung disease by 60% and the number of deaths by 15%. ”

Recently, the Trikafta received priority review status and could therefore be approved within six months by the Canadian Ministry of Health. The next step will be price negotiations between the pharmaceutical company and the provinces.

“This is great news and it’s very encouraging. The first Canadian patients could have access to Trikafta in 2021 and we can expect full coverage in 2022, ”said Dr. John Wallenburg, director of scientific activities at Cystic Fibrosis Canada.

Sherbrookoise Joanie Lévesque is treated with the drug Symdeko. “It’s like the Trikafta’s baby,” she said. I am already feeling the benefits, I have not been hospitalized this year, whereas normally I am every four months. I can’t imagine how much the Trikafta will improve our quality of life! It is almost unexpected, the drug exists and we do not have access to it. It’s inhumane.”

Only 5% of people with cystic fibrosis are receptive to Symdeko, while Trikafta is for 90% of patients.

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