Newly-approved drug called ‘significant breakthrough’ for cystic fibrosis

AUSTIN (KXAN) — The FDA has approved a new drug for the treatment of cystic fibrosis (CF), a medicine people in the pharmaceutical community are calling a “significant breakthrough.”

The drug, TRIKAFTA, was approved for people ages 12 years and older and is based on two positive global Phase 3 studies. Doctors say this has the potential to treat up to 90% of all people with CF in the future.

“[This] 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,” said Steven Rowe, M.D., the director of the Gregory Fleming James Cystic Fibrosis Research Center at the University of Alabama at Birmingham.

CF is a rare genetic disease known to shorten lifespans and impacts internal organs including the lungs, liver, sinuses, sweat glands, pancreas and reproductive tract, experts say. It affects 75,000 people worldwide with a median age of death in the early 30’s.

On KXAN News at 9 and 10 p.m., Alex Caprariello will interview Mac Rung, a UT freshman who was on the clinical trials for TRIKAFTA. He’ll share the big improvements he felt through its use.