Business Wire·Jun 5, 2026, 12:00 PM ET

Vertex Presents New Data on ALYFTREK® at European Cystic Fibrosis Conference

VRTX

Vertex announced Phase 3 data for ALYFTREK in children ages 2 to 5 with vanzacaftor/tezacaftor/deutivacaftor-responsive genotypes, showing 65% achieved sweat chloride levels below 30 mmol/L.
Vertex plans to initiate global regulatory submissions for ALYFTREK in children ages 2 to 5 during the first half of 2026.
96-week interim analyses from open-label extension studies demonstrate a positive long-term safety & efficacy profile for ALYFTREK in individuals ages 6 and older.
Vertex presented Phase 3 data for TRIKAFTA in children ages 1 to <2 years & has already initiated global regulatory submissions for this age group.
In the 24-week Phase 3 study for ALYFTREK in children ages 2 to 5, 92% of participants achieved sweat chloride concentrations below 60 mmol/L.
Treatment with TRIKAFTA in children ages 1 to <2 resulted in a mean sweat chloride reduction of 71.8 mmol/L, with 68.6% reaching levels below 30 mmol/L.
Both ALYFTREK & TRIKAFTA carry warnings for drug-induced liver injury & liver failure, requiring frequent liver function test monitoring.
Warnings for both medicines include hypersensitivity reactions, intracranial hypertension, neuropsychiatric events, & potential drug interactions with CYP3A inducers or inhibitors.
Cataracts have been reported in pediatric patients treated with TRIKAFTA, necessitating baseline & follow-up ophthalmological examinations.
ALYFTREK is currently indicated for patients ages 6 and older, while TRIKAFTA is indicated for patients ages 2 and older; use in younger children remains investigational.
Vertex stated these findings support the mission to restore CFTR function in patients with cystic fibrosis as early as possible in the disease course.