Encouraging results from study of cystic fibrosis drug for children ages 6 to 11

A potential new cystic fibrosis treatment for ages 6 to 11 showed encouraging results, improving lung function and reducing the key measure of the disease’s severity.

A potential new cystic fibrosis treatment for children ages 6 to 11 showed encouraging results, improving lung function.

The three-drug mixture known as Trikafta (a combination of elexacaftor, ivacaftor and tezacaftor) is already approved by the Food and Drug Administration for use in kids 12 and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR). About 90% of people with cystic fibrosis are estimated to have the particular mutation.

The latest trial for safety and effectiveness for younger patients showed the treatment generally had the same positive results as experienced by older patients. Lung function in the younger patients — measured as the percent predicted forced expiratory volume in one second (ppFEV1) — improved 10 percentage points after nearly six months of treatment.

“These are very exciting developments in testing the safety and efficacy of these drugs in younger children,” said Scott G. Bickel, M.D., pediatric pulmonologist with Norton Children’s Pulmonology, affiliated with the UofL School of Medicine. “We’ve already seen impressive results from the drug in older children.”

The Food and Drug Administration already is considering a supplemental new drug application for Trikafta in children 6 to 11 and assigned it priority review.

Recognized for high-quality cystic fibrosis care

The Norton Children’s Cystic Fibrosis Program has been recognized by the Cystic Fibrosis Foundation for “comprehensive, high-quality care.”

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The most common reported side effects of the treatment included mild to moderate cough, headache and fever. The treatment sometimes increases cough for several days as old, built-up mucus is “purged” from the airways. After a few days, the cough and baseline cystic fibrosis respiratory symptoms typically improve significantly.

Cystic fibrosis is a rare, progressive genetic disease. In patients with cystic fibrosis, the amount of water and salt in many areas of the body is unbalanced. As a result, cellular surfaces can’t absorb water, causing thick and sticky mucus that clogs various passageways in the lungs, digestive tract and other parts of the body. It leads to severe respiratory and digestive issues.

Trikafta targets the defective CFTR protein to help it function more effectively and thus partially reverse the underlying issue that causes cystic fibrosis.


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