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 patients with cystic fibrosis (CF) with certain mutations between 6-11 years of age.

The approval, granted to Vertex Pharmaceuticals Incorporated, provides for the first time ever a treatment option for approximately 1,500 cystic fibrosis patients in this age group with at least 1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that responds to treatment in vitro.

“Clinical experience with TRIKAFTA in patients 12 years and older over the past 20 months has demonstrated that this drug 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 being able to now treat younger patients with this breakthrough drug, including those who have not shown significant signs of disease progression.

“In addition to bringing TRIKAFTA to a younger patient population, patients previously ineligible for a CFTR modulator will now have access to 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 aged 6-11 years who have either 2 copies of the F508del mutation or 1 copy of the F508del mutation and 1 minimal function mutation .

The treatment was considered well tolerated, with a safety profile comparable to that found in previous studies in patients 12 years of age and older.

The drugs

The drug is a combination of 3 drugs that target the defective CFTR protein by making 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 in the lungs, digestive tract, and other parts of the body leading to serious 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 drugs are currently available to address the defective protein, many patients with cystic fibrosis have mutations that are ineligible for treatment.

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

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

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