Vertex gets Canada OK for cystic fibrosis treatment

-SYMDEKO is Vertex’s third medicine to treat the underlying cause of CF-

-Approximately 2,000 people in Canada are ages 12 and older and have two copies of the F508del mutation or at least one mutation in the CF gene that is responsive to treatment with SYMDEKO-

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada approved ^PrSYMDEKO™ (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,” said 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.”

Approval was based on data from two Phase 3 studies (EVOLVE and EXPAND), 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 treated with SYMDEKO experienced statistically significant improvements in lung function, as determined by absolute change from baseline in percent predicted forced expiratory volume in one second (ppFEV₁). The treatment was generally well tolerated; the most common adverse reactions (≥10%) experienced by patients who received SYMDEKO in the pooled, placebo-controlled Phase 3 studies 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 earlier this month add to the growing body of evidence supporting the benefit of long-term treatment of the underlying cause of the disease. The analysis demonstrated a safety profile consistent with that 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,” said 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 percent 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 F508delmutation, 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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