Canada Approves Trikafta for Children 6 and Older

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Health Canada has expanded the approval of Vertex Pharmaceuticals‘ triple-combination modulator treatment Trikafta to cover children with cystic fibrosis (CF) as young as 6 who have at least one copy of the F508del mutation.

Trikafta had been approved only for patients ages 12 and older in Canada. According to Vertex, about 500 children in Canada now will be eligible for the treatment. Vertex also is making available a new dosage tablet of Trikafta (elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor 75 mg) to facilitate its use in these young patients.

“We are delighted that TRIKAFTA is now available for these young patients in Canada. It provides a new treatment option for those with CF ages 6–11 with at least one F508del mutation and a first-in-class treatment option for the approximately 500 6–11-year-olds who are newly eligible for a medicine that treats the underlying cause of their disease,” Reshma Kewalramani, MD, Vertex’s president and CEO, said in a press release.

Trikafta contains a combination of three CFTR modulators, medications that help to correct the functionality of the CFTR protein in people with specific CF-causing mutations. F508del is the most common; about 90% of CF patients have carry at least one copy of this mutation.

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The Health Canada approval, which follows a similar decision in the U.S. last year, was supported by data from clinical trials that showed Trikafta to be safe and effective in children as young as 6.

“This important milestone brings us one step closer to our ultimate goal of developing treatments for all patients living with CF. We will now work closely with all provinces and territories to secure access for eligible patients as quickly as possible,” Kewalramani said.

Cystic Fibrosis Canada celebrated the approval, and called on public and private insurance entities in Canada to provide coverage for Trikafta.

“Cystic fibrosis is a progressive disease, and children accumulate extensive damage to their lungs between the ages of six and 20. This damage is often irreversible and causes severe harm to their overall health and wellbeing. Early access to Trikafta can slow the progression of symptoms and reduce the decline in a child’s condition, forever altering the course of their disease and their future,” John Wallenburg, PhD, chief scientific officer of Cystic Fibrosis Canada, said in a separate press release.

The Canadian Agency for Drugs and Technologies in Health (CADTH) and Quebec’s Institut national d’excellence en santé et en services sociaux (INESSS), agencies that help interpret medical information for decision-makers in Canada, are expected to update their recommendations on Trikafta’s cost-effectiveness to include patients as young as 6.

When CADTH and INESSS had reviewed Trikafta after its initial Canadian approval last year, the agencies recommended that the therapy should be used only by patients whose lung function was lower than 90% than of predicted capacity, and that patients need to show evidence of marked lung function improvement to continue treatment. Cystic Fibrosis Canada has criticized these recommendations as needlessly restrictive.

“Trikafta is already working miracles for many people aged 12 and over in Canada,” Wallenburg said. “We need to ensure that no one is left behind. Private and public funders should not impose clinical conditions. Clinicians — and only clinicians — should be making treatment decisions for their patients.”