Vertex announces positive results from trial for cystic fibrosis combination treatment

Betsy Goodfellow | February 6, 2024 | News story | Research and Development |  Chronic Diseases, Vertex pharmaceuticals, clinical trial, combination treatment, cystic fibrosis 

Vertex Pharmaceuticals has announced positive results from its once-daily vanzacaftor/tezacaftor/deutivacaftor programme, the company’s phase 3 programme for the treatment of cystic fibrosis (CF).

The programme included two randomised, double-blind, active-controlled, 52-week trials, SKYLINE 102 and SKYLINE 103, which assessed the efficacy of the drug combination in patients with CF aged 12 and older who have at least one F508del mutation or mutation responsive to triple combination CFTR modulators (CFTRm), compared to Trikafta. A third phase 3, single-arm, 24-week, open-label study, RIDGELINE 105, assessed the safety and efficacy of the drug combination in children with CF between the ages of six and 11, with at least one mutation responsive to triple combination CFTRm.

Carmen Bozic MD, executive vice president, global medicines development and medical affairs, and chief medical officer at Vertex, commented: “We are very pleased with today’s results, which demonstrate the vanza triple is non-inferior to TRIKAFTA in improving lung function and superior to TRIKAFTA in lowering levels of sweat chloride in people living with CF, setting a new standard for the level of CFTR protein function achievable, and raising the very high bar set by TRIKAFTA. We look forward to submitting our application to regulators with the aim of bringing this potential medicine to patients as quickly as possible.”

Betsy Goodfellow