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Volunteer Advocate

Trikafta

Trikafta is a transformational drug that can 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?

On Friday, June 18th, 2021, Health Canada approved Trikafta for sale in Canada for people aged 12 and up with cystic fibrosis and at least one F508del mutation.

Every province and territory in Canada has indicated they have or will fund Trikafta for eligible people living with cystic fibrosis. These funding announcements do not always equal access for all who could benefit from the drug. Some jurisdictions have restrictions on access and complex reimbursement systems. Cystic Fibrosis Canada is advocating for improved access to this transformational drug for all who could benefit from it. 

For updates on the accessibility of Trikafta through public funding please see our Access to Trikafta by Location page.  


TRIKAFTA RESEARCH

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.

Trikafta was fast-tracked for access by the U.S Food and Drug Administration (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 numerous countries including: the United States, United Kingdom, Ireland, Austria, Denmark, Germany, and Slovenia.


WHAT CAN I DO TO HELP GET TRIKAFTA FUNDED ACROSS CANADA?

Stand up and make your voice heard. We need a commitment from provincial governments to immediately fund Trikafta. Ways that you can help:


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 12 with one copy of the F508del mutation, regardless of their second mutation.

The manufacturer has submitted an extension for children 6-11 to Health Canada. 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.  

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 companies (AbbVie and Vertex Pharmaceuticals) are developing other potential triple combination therapies.  

Please see our clinical trial finder webpage for opportunities to be involved in trials for future CF therapies.