Health Canada Approves PrSYMDEKO (tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis

Genetics Investing

Vertex Pharmaceuticals (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 …

Vertex Pharmaceuticals (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: P67LD110HR117CL206WR352QA455ED579G711+3A→GS945LS977FR1070WD1152H2789+5G→A3272-26A→G, and 3849+10kbC→T.

As quoted in the press release:

“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).

Click here to read the full press release.

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