Vertex reports interim ALYFTREK pancreatic-function data in young children with cystic fibrosis
Vertex Pharmaceuticals released interim findings from an ongoing study of ALYFTREK in children aged 2 to 5 with cystic fibrosis. Among 48 children in a PERT-discontinuation substudy, 18, or 37.5%, stopped pancreatic enzyme replacement therapy for a mean of 8.6 weeks; across the 66-child cohort, 50% were not receiving PERT at the data cut. Mean fecal elastase-1 rose by 110.2 μg/g to 239.7 μg/g, and 48.6% of children reached the study's pancreatic-function benchmark. Vertex shared the findings at NACFC in Atlanta, while ALYFTREK remains investigational for this age group.
Why it matters
The findings could support future expansion of ALYFTREK into younger cystic-fibrosis patients, but use in children aged 2 to 5 is not yet approved. The reported ability of some participants to stop PERT may strengthen the case for further regulatory and clinical development, although these are interim data.
Key facts
- 1The interim analysis covered 48 children in a PERT-discontinuation substudy and 66 children in the full enrolled cohort. finanznachrichten.de
- 218 children, equal to 37.5%, stopped PERT for a mean of 8.6 weeks at the interim analysis, while 7 others had stopped before entering the substudy. finanznachrichten.de
- 3Mean fecal elastase-1 reached 239.7 μg/g, an increase of 110.2 μg/g from baseline. finanznachrichten.de
- 4Across the 66-child cohort, 50% were not receiving PERT at the data cut. finanznachrichten.de
- 5ALYFTREK use in children aged 2 to 5 with cystic fibrosis remains investigational. finanznachrichten.de
Summary written by AlphAI AI Desk from 3 of 3 sources. Not investment advice. Figures are as stated by the linked sources.