Trikafta Soon Be Up for Approval, Cystic Fibrosis Canada Reporting

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by David Melamed, PhD |

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It's only 2 miles to Canada along I-89 north of Burlington, Vermont. (Photo by Larry Luxner)

Following yearlong advocacy efforts, the triple-combination therapy Trikafta is expected to soon come up for approval before Health Canada, the country’s health regulatory agency, the non-profit patient group Cystic Fibrosis Canada has announced on its website.

Vertex Pharmaceuticals, which developed and markets Trikafta, its newest modulator therapy for cystic fibrosis and one able to treat up to 90% of all patients, has decided “to bring new CF medicines to Canada,” the group’s release stated.

“We understand this to mean that an application to bring Trikafta to Canada is underway” although not yet submitted, it continued, noting a Vertex application is not listed on the Health Canada webpage for drugs under review.

Once submitted, the country’s health minister has “committed to fast track Trikafta through Health Canada for approval and for pricing review,” the release added.

This announcement follows efforts by Cystic Fibrosis Canada, others in the community like CF Get Loud and partner groups, advocating for Trikafta’s availability in Canada and raising awareness of issues with its reimbursement system for rare disease therapies. These efforts included flooding members of Parliament with over 5,500 letters, organizing an open letter signed by 77 doctors with a CF specialty, and a petition signed by 40,000 Canadians.

Meetings with government officials were also held.

“This is an extraordinary moment of hope for people living with or impacted by cystic fibrosis in Canada, earned through the relentless work of our community,” said Kelly Grover, president and CEO of Cystic Fibrosis Canada.

“Thousands of letters have been sent, petitions and open letters signed, meetings held with MPs [members of Parliament], personal stories bravely shared with the media, all creating a swell of urgency that could not be ignored. Thank you to everyone who helped make this possible,” Grover added.

Trikafta combines elexacaftortezacaftor, and ivacaftor to treat CF caused by at least one F508del mutation — a mutation present in about 90% of all patients.

Mutations to the CFTR gene that codes for the CFTR protein, which allows for the transport of salts across cell membranes of organs that include the lungs and pancreas, cause the disease. These mutations result in a defective CFTR protein, and an imbalance in salt transport that leads to disease symptoms.

The triple combination treatment is designed to raise levels of a working CFTR protein in patients’ cells.

Data based on Canadian research suggested that were Trikafta to become available in 2021, access would result by 2030 in 15% fewer deaths, 60% fewer people living with severe lung disease, and a median of 9.2 more years of life for children born with CF.

Trikafta is approved for use in the U.S. and across the European Union (where it’s being marketed under the brand name Kaftrio), and is under review in the U.K., with an opinion expected in December.

“While Trikafta has been available for over a year in the United States, the manufacturer had not submitted an application to bring the drug to Canada, citing uncertainty caused by changes to the Patented Medicine Prices Review Board (PMPRB) guidelines that inform the regulation of patented drug pricing” in the country, Cystic Fibrosis Canada stated in its release.

Once the application is filed and approved by Health Canada, it moves to the pan-Canadian Pharmaceutical Alliance (pCPA) that negotiates drug pricing directly with manufacturers.

The group is asking pCPA members to include Trikafta in ongoing discussions with Vertex concerning that company’s two other CF treatments, Orkambi (lumacaftor/ivacaftor) and Kalydeco (ivacaftor).

“We want to point out that this did not come as quickly as it should have and we lost members of our community in the process,” said John Wallenburg, chief scientific officer at Cystic Fibrosis Canada. “We call on all levels of government to move this drug through the approval process quickly so people living with cystic fibrosis do not have to wait any longer.”

The group also asks that people interested in continuing to push for Trikafta’s availability in Canada join its National Advocacy Network, among other advocacy steps open to the public.


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