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Cystic fibrosis is a complex disease caused by mutations in the gene for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). There are over 2,090 known mutations. CFTR modulators, target the cause of cystic fibrosis by correcting the defective CFTR protein, whereas other CF treatments and therapies manage the symptoms of CF.
All modulators can stabilize the health and reduce the number of pulmonary exacerbations experienced by Canadians with cystic fibrosis. Highly Effective Modulator Therapies (HEMTs) like Kalydeco and Trikafta can dramatically improve the health of people with cystic fibrosis.
The health benefits of CFTR modulators Kalydeco and Trikafta in particular have been nothing short of spectacular, with patients and physicians alike describing them as life-changing. There is no cure for cystic fibrosis and CFTR modulator drugs are the closest thing that many people living with cystic fibrosis have to a cure.
Yet Canada’s system of approving and reimbursing drugs for rare diseases is cumbersome and convoluted and many people living with cystic fibrosis in Canada have been unable to access these new, life-changing drugs. Cystic Fibrosis Canada is working to change this.
Kalydeco, the first generation modulator, was approved by Health Canada for treatment of a specific mutation in 2012 and for additional mutations in 2014. Altogether Kalydeco could treat about 4% of the cystic fibrosis population in Canada. However, more than 6 years after it was first approved, Canadians still don’t have public access to Kalydeco for all the mutations it can treat.
The second-generation modulators, Orkambi and Symdeko, were approved by Health Canada in 2016 and 2018, respectively. They could treat about 50% of the Canadian cystic fibrosis population that carry two copies of the most common CF mutation, delF508.
Orkambi is publicly available in four provinces: in Quebec under the ‘patient d’exception’ program, and in Saskatchewan, Alberta, and Ontario under extremely restrictive access criteria. Symdeko may be available through some private insurance plans, as the manufacturer has thus far declined to put the medicine through Canada’s challenging drug review and reimbursement system for precision medicines.
The third-generation of modulators, Trikafta, is a new transformational drug that can treat up to 90% of Canadians with cystic fibrosis. Trikafta was fast-tracked for access by the U.S Federal Drug Agency (FDA) and the European Medicines Agency (EMA). Trikafta has received regulatory approval in 32 countries, 27 within the EMA centralized regulatory approval in addition to receiving approval in the United States, United Kingdom, Norway, Iceland and Liechtenstein. Trikafta has received public reimbursement in the United States, United Kingdom, Ireland, Austria, Denmark, Germany, and Slovenia.
Around 5% of the Canadian CF population does not have genetic mutations addressed by modulator therapies, meaning there are no modulators that can treat their disease. A number of other therapies that might help those who do not benefit from modulators are in early stages of development; these drugs use other, often genetic, approaches to correcting the defect in the CF protein. Cystic Fibrosis Canada continues to advance research into innovative, therapeutic treatments to ensure no person is left behind when it comes to life-changing therapies and treatments.
Additionally, while new modulators will help a large portion of the Canadian CF population, modulators do not constitute a cure. Even for people on modulators, the disease will continue to progress in ways that remain largely unpredictable. Until we find a cure, all individuals with CF will still require access to symptom management medicines and specialized clinical care.
Cystic Fibrosis Canada remains committed to ending CF for everyone. Find out more about our work.