Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 6 through 11 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 cystic fibrosis 12 years and older with at least one copy of the F508del mutation or one copy of a mutation that is responsive in vitro. An additional dosage strength of Trikafta tablets is now available (elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor 75 mg) in connection with this approval.

“Today’s approval is a critical milestone in our efforts to deliver medicines that help treat the underlying cause of this devastating disease as early in life as possible,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. “We can now reach approximately 1,500 newly eligible children in the U.S., and we continue to pursue approval for this expanded indication in other countries.”

Vertex completed a 24-week Phase 3 open-label, multicenter study which enrolled 66 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 to evaluate the safety, pharmacokinetics and efficacy of Trikafta. The regimen was generally well tolerated, and safety data were similar with those observed in previous studies of patients ages 12 years and older. The full data from this study were recently published in American Journal of Respiratory and Critical Care Medicine.

“Clinical experience with Trikafta in patients 12 and older over the past 20 months has demonstrated this medicine has a meaningful and unprecedented clinical benefit for patients. I look forward to now being able to treat younger patients with this breakthrough medicine, including those who have not presented major signals of disease progression,” said Terri Laguna M.D., M.S.C.S., Associate Director of the Cystic Fibrosis Center and Division Head, Pulmonary and Sleep Medicine, Ann & Robert H. Lurie Children’s Hospital of Chicago. “In addition to bringing Trikafta to a younger patient population, patients not previously eligible for any CFTR modulator will now be able to access a treatment that targets the underlying cause of their disease.”

Trikafta? is already approved for the treatment of patients with CF ages 12 years and older with certain mutations in the U.S. Switzerland, Australia and Israel, as well as in the EU and the U.K. as KAFTRIO? (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with KALYDECO? (ivacaftor). Vertex has submitted applications for use of TRIKAFTA/KAFTRIO in children ages 6 through 11 years to the European Medicines Agency (EMA) and the Medicines & Healthcare products Regulatory Agency (MHRA) and plans to file for this expanded use in Switzerland, Australia and Israel this year.

About Cystic Fibrosis

Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 80,000 people globally. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.