TouchRESPIRATORY got the opportunity to catch up with Dr Jordana E Hoppe (Children’s Hospital Colorado, University of Colorado School of Medicine, Aurora, CO, US) to discuss the phase 3 extension study of lumacaftor–ivacaftor therapy in young children with cystic fibrosis homozygous for the F508del-CFTR mutation.
Questions
- What are the unmet needs in the treatment of young children (aged 2–5 years) with cystic fibrosis? (0:18)
- What have Phase 3 study data taught us about the efficacy and safety of CFTR modulator therapy in young children with cystic fibrosis? (2:24)
- Could you tell us a little about the Phase 3 extension study and its findings? (5:57)
- What is the likely future role of lumacaftor–ivacaftor in cystic fibrosis therapy? (7:19)
- What other therapies are in the pipeline for young children with cystic fibrosis? (8:31)
Disclosures: Jordana E Hoppe was the site principal investigator at Children’s Hospital Colorado for the study discussed in this presentation.
Support: Interview and filming supported by Touch Medical Media. Interview conducted by Victoria Jones.
Reference: Hoppe JE, Chilvers M, Ratjen F, et al. Long-term safety of lumacaftor–ivacaftor in children aged 2–5 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a multicentre, phase 3, open-label, extension study. The Lancet Respiratory Medicine. 2021; DOI: 10.1016/S2213-2600(21)00069-2.
