'It really is a miracle drug': Brantford mom calls newly approved cystic fibrosis drug life-changing
A new drug that treats people with cystic fibrosis was recently approved for funding by the provincial government.
On Friday, Ontario announced it will cover the cost for Trikafta, a gene modifying treatment for people with cystic fibrosis.
The drug roughly costs $300,000 a year.
“I’m still a little bit numb. I can’t quite process it,” said Danielle Weil, whose three-year-old daughter Emme has the genetic disorder. “It really is a miracle drug.”
Emme was diagnosed with cystic fibrosis at 12 days old. Weil said since her daughter's diagnosis, she has been fighting to have Trikafta approved in Canada.
“It’s been a long fight, but I didn’t expect it to fall into place this quickly,” Weil said.
But Weil said the fight is not over just yet. Her daughter does not qualify for the drug, as it’s only available for those 12 and older whose lungs function at 90 per cent or less.
“It’s still a bit of a sore spot for us. Like we’re saying 'why, why is this necessary?'" Weil said. “I don’t think the team of advocates in Ontario is going to let it slide.”
Cystic Fibrosis Canada said the lung function criteria was a recommendation by the Canadian Agency for Drugs and Technologies in Health but said most countries who approved Trikafta don’t have those criteria.
“It’s our recommendation to the Ontario government and really every government in Canada, that they follow, or enable, or empower our clinicians to be able to determine who should be on this drug,” said Kim Steele, director of government and community relations for Cystic Fibrosis Canada.
Cystic Fibrosis Canada calls Trikafta the biggest innovation in cystic fibrosis treatment. It says the drug could be effective in 90 per cent of patients.
“This drug treats the basic defect in the therapy, so it treats the gene for cystic fibrosis,” Steele explained. “[Trikafta] is not a cure, it doesn’t cure the gene, but it does significantly seem to slow the progression of the disease.”
Weil said she’s confident that once her daughter gets on Trikafta it will give her a healthier and longer life.
“I think if we can get her onto it young enough, these kids will have a completely normal life spam. For us, it means she going to be able to grow up with her twin sister," she said.