Vertex wins FDA approval for cystic fibrosis combo treatment

Oct. 22, 2019

The U.S. FDA has approved Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis in people ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation.

"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, Vertex's chairman, president and CEO. "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."

According to Steve Rowe, director of the Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, the FDA's approval is historic for cystic fibrosis care as it has the potential for more people to benefit from CFTR modulator therapy to treat the basic defect of their disease.

Vertex has priced its three-med combo at $311,503 per year.

Read the full Vertex release