Trikafta is a transformational drug that has the potential to treat up to 90% of Canadians with cystic fibrosis. It is a triple combination precision medicine (ivacaftor, tezacaftor and elexacaftor). Rather than just treating symptoms, Trikafta targets the basic defect from specific genetic mutations that cause CF.
IS TRIKAFTA AVAILABLE IN CANADA?
As of September 13, 2022, Trikafta is publicly funded by all provinces, territories and federal drug programs across Canada for those aged six and older with at least one F508del mutation.
For updates on the accessibility of Trikafta through public funding please see our Access to Trikafta by Location page.
Canadian research published in the Journal of Cystic Fibrosis demonstrates that access to Trikafta in 2021 would result in profound health benefits for Canadians living with cystic fibrosis. By 2030, Trikafta could reduce the number of people living with severe lung disease by 60% and reduce the number of deaths by 15%.
The findings show a significantly slower disease progression with an 18% increase in people with mild lung disease and 19% fewer hospitalizations or home intravenous antibiotics for pulmonary exacerbations. The estimated median age of survival for a child born with cystic fibrosis would increase by 9.2 years. Read more about this research.
TECHNICAL INFORMATION ON TRIKAFTA
How does Trikafta work?
Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies, such as Trikafta, are designed to correct the malfunctioning protein made by the CFTR gene. The CFTR protein regulates the proper flow of water and chloride in and out of cells lining the lungs and other organs.
In people with CF, mutations in the CFTR gene result in either a defective protein being produced or no protein at all. This leads to the buildup of thick, sticky mucus, which can lead to infections in the lungs and damage to the pancreas. It can also lead to problems in other parts of the body.
Because different mutations cause different defects in the protein, the CFTR modulators that have been developed so far are effective only in people with specific mutations.
Who can potentially benefit from Trikafta?
Health Canada approved Trikafta for use in anyone living with cystic fibrosis over the age of 6 with one copy of the F508del mutation, regardless of their second mutation.
The hope is that it will also be indicated for younger children if the necessary clinical trials prove it safe and effective in this age group – meaning that it could one day potentially benefit 90% of people living with CF in Canada.
Is Trikafta more effective than existing cystic fibrosis gene modulator therapies?
Yes, and significantly so. On average, Trikafta use leads to a 14% increase in lung function over the untreated baseline. In clinical trials of the triple combination therapy, people with two copies of the F508del mutation had a 10% increase in lung function on average compared to treatment with Symdeko (that already provides a 4% increase over baseline), and people with a single copy of F508del had, on average, more than a 14% increase in lung function compared to treatment with the placebo.
WILL EVERYONE WHO TAKES TRIKAFTA SEE SIGNIFICANT IMPROVEMENT
Cystic fibrosis is a heterogenous disease and individual responses to Trikafta will vary. Many people on Trikafta have experienced a dramatic increase in lung function while others have experienced a more moderate increase or no significant change in lung function. As a heterogenous disease, the response to Trikafta will be individual and as such, those who demonstrated a more muted improvement in lung function may have seen improvements in other areas that are equally as important to consider, such as in BMI or overall quality of life.
It is also important to note that some people may feel side effects while taking Trikafta, including rash, headache, changes to their mental health and abdominal pain. In all cases, Cystic Fibrosis Canada encourages patients to discuss how they are responding to Trikafta with their clinics.
How much does it cost?
While Trikafta is a high value drug, it is also a high cost drug. However, what public and private insurers pay is negotiated with the company.
A body called the pCPA (pan-Canadian Pharmaceutical Alliance) negotiates on behalf of the provincial, territorial and federal programs the price they will actually pay, and each jurisdiction can further negotiate with the manufacturer from there. Learn more about Canada’s drug review process.
Cystic Fibrosis Canada is advocating strongly for a drug pricing system that enables faster access to CF medicines and expedited negotiations for access to Trikafta. Find out more about our advocacy work and how you can help.
Are there any other therapies like this?
While there are other CFTR modulators available in Canada, Trikafta is the first triple combination CFTR therapy to be approved by Health Canada. At this time at least two other companies (AbbVie and Sionna Therapeutics) are developing CFTR modulator therapies and the manufacturer of Trikafta, Vertex Pharmaceuticals, continues to develop its line of CF therapeutics.
Please see our clinical trial finder webpage for opportunities to be involved in trials for future CF therapies.