A new treatment for cystic fibrosis

BOSTON—Vertex Pharmaceuticals Incorporated has announced the U.S. Food and Drug Administration (FDA) approval of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in people ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

“Today marks a milestone for CF patients, their families and Vertex. After a 20-year journey together, we have received FDA approval of Trikafta: a single breakthrough medicine with the potential to treat up to 90% of all people with CF in the future,” said Jeffrey Leiden, M.D., Ph.D., Vertex’s chairman, president and chief executive officer. “For approximately 6,000 people with CF in the U.S., Trikafta is the first medicine that can treat the underlying cause of their disease. I want to personally thank the hundreds of Vertex scientists who have been working on this program for nearly 20 years – many of whom have dedicated their entire careers to changing the course of this disease; the CF Foundation which has provided support, encouragement and help throughout the journey; and most importantly the thousands of patients, caregivers, doctors and advocates who have courageously and persistently worked side-by-side with us to get to where we are today.”

With this approval approximately 6,000 people with CF ages 12 years and older who have one F508del mutation and one minimal function mutation (F/MF) will, for the first time, have a medicine that targets the underlying cause of their CF. Approximately 12,000 people with one or two F508del mutations who are currently eligible for one of Vertex’s three other FDA-approved CF medicines will also now be eligible for Trikafta.

“Today’s approval is a historic moment in cystic fibrosis care, with the potential for more people to benefit from CFTR modulator therapy to treat the basic defect of their disease,” noted Steven Rowe, M.D., director, Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham. “In clinical trials, Trikafta was generally well tolerated and demonstrated improvements in multiple outcome measures in CF, including improvements in FEV₁, improvements in respiratory symptoms and, in the 24-week F/MF study, a reduced rate of pulmonary exacerbations and improvements in BMI.”

Trikafta is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. The approval of Trikafta was supported by positive results of two global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study in 403 people with one F508del mutation and one minimal function mutation (F/MF), and a 4-week Phase 3 study in 107 people with two F508del mutations (F/F).

“The incredible speed of this approval underscores our shared sense of urgency with the FDA and the CF community for bringing this medicine to eligible people with CF, particularly those without a medicine targeting the underlying cause of their disease. We remain committed to relentlessly pursuing the development of transformative therapies for all people living with this disease,” added Reshma Kewalramani, M.D., executive vice president, Global Medicines Development and Medical Affairs and chief medical officer at Vertex.

Vertex has submitted a Marketing Authorization Application to the European Medicines Agency (EMA) for the elexacaftor/tezacaftor/ivacaftor combination regimen. Vertex is currently evaluating elexacaftor/tezacaftor/ivacaftor in people ages 6 through 11 with F/MF and F/F CF mutations in an ongoing Phase 3 study, and plans to evaluate elexacaftor/tezacaftor/ivacaftor in children under 6 years of age as a part of planned future studies.