Public health agency the US Food and Drug Administration announced on Monday that it authorised the marketing of the first triple combination therapy Trikafta (elexacaftor/ivacaftor/tezacaftor) for the treatment of patients with the most common cystic fibrosis mutation under its Priority Review, Fast Track, Breakthrough Therapy Designation and Rare Pediatric Disease Priority Review.

The approval of Trikafta was granted to Vertex Pharmaceuticals Incorporated.

Cystic fibrosis, a rare, progressive, life-threatening disease, results in the formation of thick mucus that builds up in the lungs, digestive tract and other parts of the body. It leads to severe respiratory and digestive problems as well as other complications such as infections and diabetes. Cystic fibrosis is caused by a defective protein that results from mutations in the CFTR gene.

Trikafta is a combination of three drugs that target the defective CFTR protein. It helps the protein made by the CFTR gene mutation function more effectively. Trikafta is the first approved treatment that is effective for cystic fibrosis patients 12 years and older with at least one F508del mutation, which affects 90% of the population with cystic fibrosis or roughly 27,000 people in the US, said the agency.

The efficacy of Trikafta in patients with cystic fibrosis aged 12 years and older was demonstrated in two company trials. The first trial was a 24-week, randomized, double-blind, placebo-controlled trial in 403 patients who had an F508del mutation and a mutation on the second allele. The second trial was a four-week, randomized, double-blind, active-controlled trial in 107 patients who had two identical F508del mutations.

In each trial, the primary analysis looked at increases in the percent predicted forced expiratory volume in one second, called ppFEV1, which is an established marker of cystic fibrosis lung disease progression. Trikafta increased the ppFEV1 in both trials, concluded the agency.