Vertex Announces U.S. FDA Approval for TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 2 Through 5 With Certain Mutations

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved the expanded use of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 2 through 5 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive to TRIKAFTA based on in vitro data. TRIKAFTA was previously approved by the FDA for use in people with CF 6 years and older with at least one F508del mutation or a mutation in the CFTR gene that is responsive to TRIKAFTA based on in vitro data.

“Since its initial approval, TRIKAFTA has had a significant impact on the CF community, transforming the lives of thousands of people living with cystic fibrosis,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex. “We remain steadfast in our commitment to bringing highly effective CF treatments to people of all ages living with this disease.”

This label expansion was supported by a 24-week Phase 3 open-label study which enrolled 75 children ages 2 through 5 years old with CF to evaluate the safety, pharmacokinetics and efficacy of TRIKAFTA. The regimen was generally well tolerated, with a safety profile consistent with that observed in older age groups, and led to improvements in sweat chloride concentration, a measure of CFTR function, and lung function. The data from this study were recently published in the American Journal of Respiratory and Critical Care Medicine.

“Early intervention with CFTR modulator therapies like TRIKAFTA can offer the potential to improve the trajectory of CF lung disease,” said Jennifer Goralski, M.D., Assistant Professor of Medicine and Pediatrics, Co-Director, Adult Cystic Fibrosis Center, University of North Carolina School of Medicine, and a lead Principal Investigator in the TRIKAFTA 2- to 5-year-old pivotal clinical trial. “With this approval, we now have the ability to treat young children with TRIKAFTA and can proactively address the underlying cause of their disease.”

TRIKAFTA was previously approved for the treatment of people with CF ages 6 years and older with certain mutations in the U.S., Canada, Switzerland, Australia, New Zealand and Israel, as well as in the EU, the U.K., Iceland, Liechtenstein and Norway as KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with KALYDECO (ivacaftor). Additionally, Vertex has submitted applications for the use of TRIKAFTA/KAFTRIO in children ages 2 through 5 years of age to other global regulatory authorities, including the European Medicines Agency (EMA) and the Medicines and Healthcare Products Regulatory Agency (MHRA).