Children aged 2 to 5 with cystic fibrosis who switched from Trikafta to the newer once-daily drug Alyftrek saw their sweat chloride levels fall further, with about two-thirds reaching the range considered normal. Vertex Pharmaceuticals presented the data as a late-breaking abstract at the European Cystic Fibrosis Conference on June 5.
The design matters as much as the result. This was a 24-week, open-label safety and tolerability study in 67 children, all of whom were already taking an existing triple-combination CFTR modulator and had genotypes expected to respond, including two copies of F508del or F508del with a minimal function mutation. There was no placebo group and no randomization, so the changes observed cannot be attributed to the new drug with certainty. Alyftrek is approved in the United States for patients 6 and older. Its use in children 2 to 5 remains investigational.
