Vertex wins FDA label expansion for cystic fibrosis therapy Trikafta
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Vertex Pharmaceuticals (NASDAQ:VRTX) announced Wednesday the FDA approval for its cystic fibrosis therapy Trikafta to include children aged 2 through 5 years.
The CFTR modulator was previously indicated for CF patients aged six years and older with certain CF-linked gene mutations: F508del mutation or a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The label expansion is backed by data from a 75-patient Phase 3 open-label study which showed the safety and efficacy of Trikafta over 24 weeks.
CF is a genetic disorder with a systemic impact on multiple organs, including the lungs, liver, pancreas, GI tract, and reproductive tract. The rare condition is estimated to affect more than 88,000 people globally, with many indicating at least one F508del mutation.


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