Vertex announced that the Food and Drug Administration (FDA) has approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for the treatment of the underlying cause of cystic fibrosis (CF) in patients aged ≥12 years who have 2 copies of the F308del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or who have ≥1 mutation that is responsive to tezacaftor/ivacaftor. 

Symdeko was evaluated in two Phase 3 studies, EVOLVE and EXPAND, involving 750 patients with CF. In both studies, Symdeko-treated patients demonstrated statistically significant and clinically meaningful improvements in lung function and other disease outcomes. 

Treatment with Symdeko also resulted in a favorable safety profile; infective pulmonary exacerbation and cough were the most common adverse events seen across both treatment arms. Preliminary data from the ongoing EXTEND rollover study showed that lung function improvements and the safety and tolerability profiles seen in EVOLVE and EXPAND were sustained for up to 48 weeks of treatment. 

Principal investigator of the EXTEND study, Patrick Flume, MD, added, “In particular, Symdeko is an important treatment option for patients who either never started or discontinued Orkambi, and it also provides increased benefit over Kalydeco alone for patients with residual function mutations.”

Symdeko will be available starting this week. It is co-packaged as tezacaftor 100mg/ ivacaftor 150mg fixed dose combination tablets and ivacaftor 150mg strength tablets in 56-count cartons (4-week supply). 

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