A year out from FDA approval, Vertex looks to expand top-selling CF drug to pediatrics
About a year after getting the FDA thumbs-up for its blockbuster cystic fibrosis drug Trikafta, Vertex is picking up where it left off.
The Boston-based biotech read out positive results from a Phase III study involving children ages 6 to 11, and announced plans to file for a supplemental NDA later this year. Previously, the triplet regimen was only available to patients 12 and up.
Trikafta, Vertex’s $VRTX fourth CF drug, is designed for those with at least one F508del mutation in the CFTR gene. It covers about 90% of CF patients, unlike the company’s Kalydeco, which only covers 6% of Americans with CF, according to a STAT report. Within weeks of Trikafta’s October 2019 launch, it became Vertex’s top-selling drug. Quarterly US sales hit $420 million by the end of January.
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