Positive Phase 3 Study for Tezacaftor/Ivacaftor Combination in Children Aged 6-11 Years with Cystic Fibrosis Supports European Medicines Agency Submission

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the results of a Phase 3 study conducted in Europe and Australia of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis (CF) who have either two copies of the F508del mutation or one copy of the F508del mutation and one residual function mutation. The study met its primary endpoint of absolute change in lung clearance index (LCI_2.5) through 8 weeks of treatment, demonstrating a statistically significant improvement in LCI_2.5 among patients treated with tezacaftor/ivacaftor. The regimen was generally well tolerated and safety data were consistent with those observed in previous studies with tezacaftor/ivacaftor. This efficacy study was designed to support a submission to the European Medicines Agency (EMA) to extend the indication of tezacaftor/ivacaftor in this patient population. Vertex plans to submit the application in the second half of 2019. In late 2018, Vertex submitted an sNDA to the U.S. Food and Drug Administration (FDA) for tezacaftor/ivacaftor based on a previously completed Phase 3 safety study in children ages 6 through 11 years of age conducted in the U.S. and Canada.

“These data mark an important milestone in our efforts to expand treatment options for patients living with CF,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We plan to submit an indication extension to the EMA in the second half of 2019, bringing us a step closer to potentially providing more children with a treatment option that addresses the underlying cause of the disease.”

Summary of Key Data

The data announced today are from a Phase 3, randomized, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in children ages 6 through 11 who have either two copies of the F508del mutation or one copy of the F508del mutation and one residual function mutation. Subjects were randomized 4:1 based on their genotype to tezacaftor/ivacaftor versus a blinding arm (placebo for those with two copies of F508del; ivacaftor for those with one copy of F508del mutation and one residual function mutation). The study randomized and treated 54 subjects with TEZ/IVA, 10 with placebo, and 3 with ivacaftor.