Health Canada Approves Trikafta for CF Patients, Ages 12 and Up

Health Canada has approved Trikafta  as a triple-combination therapy for people with cystic fibrosis (CF), ages 12 and up, who have at least one F508del mutation, the most common CF-causing mutation.

This decision is expected to give about 1,100 people access for a first time to a therapy that directly addresses the underlying cause of their disease, according to Trikafta’s developer, Vertex Pharmaceuticals. A study drawing on Canadian data estimated that CF-related deaths across that country could drop by 15% over the next 10 years with this medication’s use at the start of this decade.

“The approval of TRIKAFTA marks a significant milestone for Canadians with CF, their families and Vertex,” Reshma Kewalramani, MD, president and CEO of Vertex, said in a press release.

“This is a special day for our community. Together we have been fighting hard for access to these medications. Thousands of letters and signatures and hundreds of meetings later, we are finally seeing the progress our community deserves,” Kelly Grover, president and CEO of Cystic Fibrosis Canada, said in a separate release.

CF is caused by mutations in the CFTR gene that codes for a protein of the same name. Trikafta is a combination of three CFTR modulators (elexacaftor, tezacaftor, and ivacaftor), which can increase the functionality of the mutated protein in people with specific mutations. F508del is the most common CF-causing mutation, with one copy found about nine in every 10 patients, or 90% of all those with CF.

In clinical trials, Trikafta has been shown to significantly improve lung function in patients who have at least one F508del mutation.

“I have seen substantial improvements in patients treated with TRIKAFTA in clinical practice, including improved lung function. I’m excited that more Canadians may be able to benefit from CFTR modulators and look forward to seeing the impact of this medicine for all patients who can benefit from it,” said Elizabeth Tullis, MD, medical director at the Toronto Adult CF Centre and a professor of medicine at the University of Toronto.

Trikafta’s approval in Canada was followed with an announcement by the pan-Canadian Pharmaceutical Alliance (pCPA), noting it has reached a negotiated price for Orkambi and Kalydeco — two other CFTR modulator therapies marketed by Vertex — for all Health Canada-approved CF mutations for these therapies. The pCPA, a regulatory body, negotiates medication prices on behalf of Canada’s 10 provinces and three territories, which run their own public health programs.

Price details coming out of these negotiations were not announced.

Health Canada’s approval is a first step in bringing a treatment into use, Cystic Fibrosis Canada noted in its release. Several other reviews and approvals — some already underway — are necessary “before Trikafta is publicly funded and accessible,” the group stated.

“We turn to the provinces next,” Grover said. “They must immediately fund Kalydeco and Orkambi, which have been in negotiations for more than a year, and fund Trikafta as soon as possible. Provinces, end the wait and save lives.

“People living with cystic fibrosis need these drugs, and they need them now,” she added. “No Canadian should have to fight this long and this hard for the medications they need. People with CF can’t wait. It’s literally a matter of life and death.”

Use of Trikafta, approved in the U.S. for patients, 12 and older, with one F508del mutation in 2019, was recently expanded to cover eligible children starting at 6 years old. The triple-combination medication is also approved for eligible patients 12 and older in Australia, and in Europe, where it is marketed as Kaftrio.

“I would like to thank the people with CF who participated in our clinical trials, our dedicated scientists and the investigators who have enabled this innovative medicine to be approved in Canada today,” Kewalramani said. “Without their commitment, this milestone would not have been possible.”