Two FDA Approvals for Expanded Pediatric Indications

Vertex Pharmaceuticals has received FDA approval to expand the use of Trikafta to include children ages 2 to 5 who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in this gene that responds to the drug based on in vitro data. Trikafta was previously approved for this purpose in people ages 6 years and older.

The approval decision was based on the results of a study aimed to evaluate the safety and effectiveness of Trikafta in younger children.

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Cystic fibrosis (CF) is a rare genetic disease affecting more than 88,000 people worldwide. Most people with CF have at least one F508del mutation.  CFTR mutations lead to defective or insufficient CFTR protein, causing poor flow of salt and water throughout the body. This impairs the function of multiple organs, especially the lungs, where the buildup of abnormally thick mucus results in chronic infections and progressive damage. The condition is typically diagnosed by age two.

The approval decision was based on the results of a 24-week Phase 3 open-label study involving 75 children with CF aged 2 to 5 years old. The study aimed to evaluate the safety and effectiveness of Trikafta in younger children. The results showed that the treatment was generally well-tolerated, with a safety profile consistent with that seen in older age groups. The study also revealed improvements in lung function and CFTR protein function, as indicated by measuring the amount of chloride in sweat.

Trikafta (a combination of elexacaftor, tezacaftor, and ivacaftor) is supplied as oral capsules or granules. It is typically taken twice a day.

According to GoodRx, the retail price for a one-month supply of Trikafta averages $30,000.

Pneumococcal vaccine Prevnar 20 now approved for infants and children

The FDA recently approved the Prevnar 20 vaccine for the following pediatric indications:

• to prevent invasive disease caused by 20 different serotypes of Streptococcus pneumoniae in children ages 6 weeks and older.
• to prevent otitis media (ear infection) caused by 7 serotypes of Streptococcus pneumoniae in children ages 6 weeks to 5 years.

Prevnar 20 is a vaccine containing 20 different serotypes of Streptococcus pneumoniae. The vaccine was initially approved by the FDA in 2021 to prevent pneumonia and invasive disease in individuals ages 18 years and older.

The bacteria Streptococcus pneumoniae causes pneumonia and invasive pneumococcal disease, such as bloodstream infections and meningitis. It is also a common cause of ear infections in children.

Most health insurance plans cover pneumococcal vaccines. Without coverage, the cost of Prevnar 20 averages around $265, as reported by GoodRx.