FDA Approves Cystic Fibrosis Treatment for More Pediatric Patients

The US Food and Drug Administration (FDA) has approved elexacaftor/tezacaftor/ivacaftor and ivacaftor (TRIKAFTA) for pediatric cystic fibrosis (CF) patients with certain mutations between 6-11 years old.

The approval, awarded to Vertex Pharmaceuticals Incorporated, will give for the first time ever a treatment option for approximately 1500 cystic fibrosis patients in this age group with who have at least 1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive to the treatment in vitro.

“Clinical experience with TRIKAFTA in patients 12 and older over the past 20 months has demonstrated this medicine has a meaningful and unprecedented clinical benefit for patients,” said Terri Laguna MD, MSCS, Associate Director of the Cystic Fibrosis Center and Division Head, Pulmonary and Sleep Medicine, Ann & Robert H. Lurie Children’s Hospital of Chicago, in a statement. "I look forward to now being able to treat younger patients with this breakthrough medicine, including those who have not presented major signals of disease progression.

“In addition to bringing TRIKAFTA to a younger patient population, patients not previously eligible for any CFTR modulator will now be able to access a treatment that targets the underlying cause of their disease.”

The Study

The approval was based on a 24-week phase 3 open-label, multicenter study involving 66 CF patients between 6-11 years old who have either 2 copies of the F508del mutation or 1 copy of the F508del mutation and 1 minimal function mutation.

The treatment was deemed well-tolerated, with a similar safety profile to what was found in previous studies of patients aged 12 years and older.

The Drug

The drug is a combination of 3 drugs that target the defective CFTR protein by helping the protein made by the CFTR gene mutation function more effectively.

Cystic fibrosis is a rare, progressive, life-threatening disease that results in the formation of thick mucus build up in the lung, digestive tract, and other parts of the body that lead to severe respiratory and digestive problems, as well as other complications including infections and diabetes.

The disease is caused by a defective protein that results from mutations in the CFTR gene.

While there are medications currently available to target the defective protein, many patients with cystic fibrosis have mutations that are ineligible for treatment.

The FDA previously approved the treatment for cystic fibrosis patients at least 12 years old with at least 1 copy of the F508del mutation or 1 copy of a mutation that is responsive in vitro.

An additional dosage strength of the tablets—elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 75 mg and ivacaftor 75 mg—are also now available.