Vertex Pharmaceuticals recently presented data from several clinical trials of cystic fibrosis treatments showing positive outcomes for CF patients.
These findings were presented at the North American Cystic Fibrosis Conference (NACFC) that ran Nov 2-4 in Indianapolis. Orkambi (ivacaftor/lumacaftor) data were shown in a poster “Safety and Efficacy of Lumacaftor/Ivacaftor (LUM/IVA) in Patients aged ?6 years with CF Homozygous for F508del-CFTR (Phase 3 Extension Study).”
It involved an analysis of an ongoing 96-week extension study (NCT03125395) of Orkambi in 240 children, age 6 to 11, with two copies of the F508del-CFTR mutation (a mutation that causes CF). An interim analysis at 48 weeks of treatment showed that Orkambi improved the patients’ lung function, sweat chloride and body mass index.