Children’s Healthcare of Atlanta pediatric pulmonologists Ajay Kasi, MD, and Rachel Linneman, MD, recently published two phase 3 clinical trial results in The Lancet Respiratory Medicine, demonstrating the effectiveness of a new drug for certain types of cystic fibrosis (CF).

Alyftrek was shown to be effective in 6–11-year-olds during a trial of 33 sites globally led by Dr. Kasi, second author on the first study. It was also shown to be effective in a phase 3 trial in those ages 12 years and older when compared to Trikafta, a related drug, in a second study co-authored by Dr. Linneman.

Overall, nine patients from Children’s participated in these clinical trials. Based on the results, the FDA approved Alyftrek, a once-daily CF transmembrane conductance regulator (CFTR) modulator, in children and adults with CF who are ages 6 and older and have the relevant mutations. Comparatively, other modulator medications need to be taken twice daily.

“This next generation medication will be available for even more individuals of various genotypes with CF and will provide an additional option for those that could not tolerate Trikafta,” says Dr. Linneman.

Cystic fibrosis is a genetic disease that affects the lungs and digestive system by making fluids and mucus thick and sticky. This causes persistent lung infections that progressively limit the ability to breathe. Children with CF also suffer from digestive problems that prevent their bodies from breaking down and absorbing food, leading to inadequate growth and nutrition.