Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced the company has completed a global Phase 3 study of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in children ages 6 through 11 years old with cystic fibrosis (CF) who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation, and based on the results will submit a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2020, with additional global regulatory submissions to follow.
“Our aim is to extend eligibility to all patients who may benefit from this transformative medicine, and the positive results from the study in children ages 6 through 11 years old allows us to take another step forward toward this goal,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “We are looking forward to filing an sNDA in the coming months and bringing TRIKAFTA to younger people with CF.”
Bringing TRIKAFTA to Children Less than 12 Years of Age
The 24-week global Phase 3 open-label study evaluated the safety and efficacy of TRIKAFTA in 66 children ages 6 through 11 years old who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation. The primary endpoint of the study was safety and tolerability, and the results showed that TRIKAFTA was generally well tolerated and the safety data were consistent with those observed in previous Phase 3 studies. In addition, clinically meaningful improvements were seen across multiple secondary efficacy endpoints, including improvements in percent predicted forced expiratory volume in 1 second (ppFEV1), sweat chloride, Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score, body mass index (BMI) and other measures through 24 weeks of treatment. The study showed the benefit-risk profile of TRIKAFTA in children with CF ages 6 through 11 years old was similar to that seen in people with CF ages 12 and older in the Phase 3 studies which have supported approval. Based on the results, Vertex will submit an sNDA to the U.S. FDA in the fourth quarter of 2020, with additional global regulatory submissions to follow.