Health Canada approves SYMDEKO™

Today (June 28, 2018), Health Canada announced its approval for the use of a new Vertex treatment to fight cystic fibrosis. This is great news for people living with CF as the results of clinical studies are very promising. You can read the Vertex press release for more information by clicking on This link.

What does that mean?

When Health Canada approves the use of a medication, it means that doctors have the right to prescribe it, anywhere in the country. However, this does not mean that the treatment will be reimbursed. It is possible that some private insurances cover the payment of the treatment. If you have such coverage, you can contact the insurer to check with them if Symdeko can be reimbursed to you. For people who are covered by the RAMQ, it's different. Before Symdeko can be reimbursed to you, the INESS must recommend that the medication be listed on the List of reimbursed medications. This is a process that can take several months. However, as with Orkambi, it may be possible, under several conditions, for the medication to be reimbursed to you thanks to the measure of Exceptional Patient. We recommend that you discuss this with the members of your cystic fibrosis clinic.

For French-speaking readers, we translated the press release. Note that this is a free translation from us, not the official version, so that you can better understand how this treatment works.

Health Canada approves PrSymdeko™ (tezacaftor/ivacaftor and ivacaftor) to treat the underlying cause of cystic fibrosis in people aged 12 and older with certain CFTR gene mutations.

BOSTON- June 28, 2018— Vertex Pharmaceuticals Incorporated announced today that Health Canada has approved PrSymdeko™ (tezacaftor/ivacaftor and ivacaftor) to treat the underlying cause of cystic fibrosis (CF) in people aged 12 years and older who have two copies of the F508del mutation in the cystic fibrosis regulatory gene (CFTR) or who have one copy of the F508del mutation and one of the following mutations in the CFTR gene: P67L, D110H, R117C, R117C, R117C, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S945L, S945L, S945L, S945L, D579G, 711+3A→G, S945L, S945L, S945L, S945L, S945L, R945F, R977F, R977F, R977F, R977F, R977F, R977F, R977F, R977F, R977F, R977F, R977F, R977F, R977F, 1077F, R1070W, R1070W, R1070W, D1152Q, A455E, D579G, 711+3A→G, S945L, S945L, S945L, S945L, S945L, S945L, S977F, S977F, S977F, S977F, F, F

“This approval is an important step in our journey to improve and expand treatment options for people living with CF,” said Reshma Kewalramani, MD, executive vice president and chief medical officer at Vertex. “We have made rapid progress in the development of multiple new medications over the past year, and we will continue to invest relentlessly in our science to treat the underlying cause of CF.”

The approval is based on data from two phase 3 studies (EVOLVE and EXPAND), published in the New England Journal of Medicine In November 2017, who recruited 744 people with CF aged 12 years and older with two copies of the F508del mutation (n=504) or with one F508del mutation and a second mutation predicted to be sensitive to tezacaftor/ivacaftor (n=244). In both studies, patients treated with SYMDEKO experienced a statistically significant improvement in lung function, as determined by the absolute change from the initial FEV1 percentage value. The treatment was generally well observed; the most common side effects (≥ 10%) observed in patients who received SYMDEKO in pooled, placebo-controlled phase 3 studies were headache (14%) and nasopharyngitis (12%). The results of an interim analysis of the 96-week PHASE 3 EXTEND study presented at the conference ofEuropean Cystic Fibrosis Society (ECFS) earlier this month add to the growing body of evidence supporting the benefits of long-term treatment of the underlying cause of the condition. The analysis demonstrated a safety profile consistent with that observed in the EVOLVE and EXPAND studies and that the initial improvements in lung function observed in the EVOLVE study were maintained for up to 48 weeks.

“Since the discovery of the cystic fibrosis gene in Canada, the CF community has been hoping for a therapy that targets the root of the disease,” said Elizabeth Tullis, MD, FRCPC, director of the cystic fibrosis clinic at Toronto General Hospital. The approval of SYMDEKO brings great hope to people living with CF and their families, and provides a new therapy for nearly 50 percent of Canadians living with CF.

SYMDEKO was approved by the U.S. Food and Drug Administration (FDA) in February this year for patients aged 12 and older who have two copies of the F508del mutation, or who have at least one mutation in the CF gene that responds to treatment with SYMDEKO. The European Medicines Agency (EMA) has validated the marketing authorization application (MAA) for the tezacaftor/ivacaftor combination. The company received approval in the European Union during the second half of 2018.

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