Health Canada approves Vertex Pharmaceuticals cystic fibrosis medication

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Vertex Pharmaceuticals Incorporated announces that Health Canada approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for treating the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or who have one copy of the F508del mutation and one of the following mutations in the CFTR gene: P67L, D110H, R117C,L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.

“This approval is an important milestone in our journey to enhance and expand treatment options for people living with CF,” says Reshma Kewalramani, M.D., executive vice president and chief medical officer at Vertex. “We have made rapid progress in developing multiple new medicines over the last year, and will continue to relentlessly invest in our science to treat the underlying cause of CF.”

Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia. It is caused by a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.

Approval was based on data from two Phase 3 studies (EVOLVE and EXPAND), and was published in the New England Journal of Medicine in November 2017 that enrolled 744 people with CF ages 12 and older with two copies of the F508del mutation (n=504) or with one F508del mutation and a second mutation predicted to be responsive to tezacaftor/ivacaftor (n=244). Across both studies, patients that were treated with Symdeko experienced statistically significant improvements in lung function, as determined by absolute change from baseline in per cent predicted forced expiratory volume in one second (ppFEV1). The treatment was generally well tolerated; the most common adverse reactions experienced were headache (14%) and nasopharyngitis (12%). Results from an interim analysis of the ongoing, 96-week EXTEND Phase 3 rollover study presented at the European Cystic Fibrosis Society (ECFS) Conference adds to the evidence that supports the benefit of long-term treatment of the underlying cause of the disease. The analysis demonstrated a safety profile consistent with what had been observed in the EVOLVE and EXPAND studies and that initial improvements in lung function (measured by absolute change in ppFEV1) observed in the EVOLVE study were sustained for up to 48 weeks.

“Ever since the discovery of the CF gene in Canada, the CF community has been hoping for a therapy that targets the root of the disease,” says Elizabeth Tullis, M.D., FRCPC, director of the Toronto Adult Cystic Fibrosis Clinic at St. Michael’s Hospital. “The approval of Symdeko brings great hope to people with CF and their families, and provides a new therapy for almost 50 per cent of Canadians living with CF.”

SYMDEKO was approved by the U.S. Food and Drug Administration (FDA) in February of this year for use in 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 is responsive to treatment with Symdeko. The European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the tezacaftor/ivacaftor combination. The company expects approval in the EU in the second half of 2018.

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