Vertex reports pancreatic function recovery in young CF patients

Vertex Pharmaceuticals reported that some young cystic fibrosis patients experienced a recovery of pancreatic function after treatment with ALYFTREK, according to interim data presented at a conference. The study included 48 children aged 2 to 5 years, with 18 (37.5%) successfully discontinuing pancreatic enzyme replacement therapy (PERT) for an average of 8.6 weeks.

An additional 7 participants had stopped PERT before the substudy. For the entire group of 66 children, 50% were not on PERT at the data cut. Pancreatic function, measured by fecal elastase-1 levels, increased by a mean of 110.2 μg/g from baseline, reaching 239.7 μg/g. Nearly half (48.6%) of children reached the pancreatic sufficiency threshold of ≥200 μg/g.

Cystic fibrosis causes pancreatic exocrine insufficiency in nearly 90% of children within their first year of life, previously considered irreversible. Vertex's Executive Vice President and Chief Medical Officer emphasized the significance of these findings, highlighting a major shift in understanding the benefits of treating cystic fibrosis in its early stages.

ALYFTREK, currently approved for patients 6 years and older with cystic fibrosis and at least one responsive CFTR gene variant, remains investigational for younger children. The company also shared additional abstracts on clinical and real-world evidence of CFTR modulators at the conference.