Manufacturer moves to bring cystic fibrosis drugs to Canada

Mark Lindsay wishes his daughter, Chantelle, were here to see the fruitions of her fight to bring transformational new therapies for cystic fibrosis to Canada.

But Lindsay knows the 23-year-old, who died of complications from the disease in February, would be elated to know that other patients may be one step closer to benefiting from the treatment she couldn’t access.

Following a standoff with federal authorities over drug-pricing regulations, Vertex Pharmaceuticals said in a statement Tuesday that it’s taking steps to bring its new cystic fibrosis medicines to Canada.

The news was “bittersweet” for Mark Lindsay, who unsuccessfully campaigned for permission to put Chantelle Lindsay on the Vertex-manufactured therapy Trikafta, which experts say could have profound impacts for 90 per cent of cystic fibrosis patients.

“We told Chantelle we’d continue the fight. And we did. And I just wish she was here,” he said.

“I’m sure she’d be looking down and smiling with her little grin, and equally as happy as us.”

The Lindsays’ loss became a rallying cry in health advocates’ prolonged campaign to help Canadians with cystic fibrosis gain access to Trikafta, which has been approved in the U.S. for about a year.

Vertex declined to explain why it delayed applying for the drug to be approved by Health Canada, but said it continues to have concerns about federal drug pricing reforms that take effect Jan. 1.

The company said it reviewed the final regulations released by the arm’s-length Patented Medicine Prices Review Board last month, and decided to move forward with its new cystic fibrosis treatments in Canada in spite of its reservations.

“We remain genuinely concerned that the PMPRB Guidelines may impact access for Canadians to new innovative medicines in the future,” the manufacturer said Tuesday.

A Vertex spokesman said any drug applications for Health Canada approval will be posted on the agency’s website.

Health Canada did not immediately respond about whether it has received an application for Trikafta. The PMPRB declined to comment on Vertex’s objections to its guidelines.

Cystic Fibrosis Canada said the development could have a “life-changing” effect for many of the estimated 4,300 people with cystic fibrosis in Canada, calling Trikafta “the greatest innovation in the history” of treatments for the fatal genetic disease.

“This development means everything for people living with cystic fibrosis,” president and CEO Kelly Grover said by phone on Tuesday.