Vertex Reports Positive Outcomes for Vanzacaftor/Tezacaftor/Deutivacaftor Cystic Fibrosis Treatment Trials

Vertex Pharmaceuticals has reported successful outcomes from its Phase 3 clinical trials for a new cystic fibrosis (CF) treatment, the vanza triple combination therapy. The medication, taken once daily, has shown to be non-inferior and in some cases superior to the current standard of care, TRIKAFTA, in improving lung function and reducing sweat chloride levels in individuals with CF.

The pivotal trials, SKYLINE 102 and SKYLINE 103, were randomized, double-blind, and active-controlled, involving patients aged 12 and older with at least one F508del mutation or another mutation responsive to the triple combination therapy. The vanza triple demonstrated its efficacy in increasing the forced expiratory volume in one second (FEV1) and significantly lowering sweat chloride (SwCl) levels, a key indicator of CFTR protein function.

In the SKYLINE trials, the vanza triple not only met the primary endpoint of improving lung function but also showed superiority in reducing sweat chloride levels compared to TRIKAFTA. The therapy was well-tolerated, with a safety profile similar to that of TRIKAFTA.

A separate study, RIDGELINE 105, focused on children aged 6 to 11 years and found that the vanza triple was safe and effective, with results suggesting that early treatment could potentially prevent disease progression. In this age group, a significant majority achieved sweat chloride levels below the diagnostic threshold for CF.

Vertex Pharmaceuticals plans to submit the vanza triple for regulatory approval globally, aiming to make the treatment available for individuals with CF aged 6 and older by mid-2024. The company intends to utilize a priority review voucher in the U.S. to expedite the review process.

The positive results from the vanza triple trials have been met with optimism by medical professionals in the field. Dr. Carmen Bozic, Vertex's Executive Vice President, and Chief Medical Officer, highlighted the potential of the new therapy to set a new standard for CFTR protein function. Dr. Bonnie Ramsey, a pediatric pulmonologist with decades of experience, expressed hope for the future of CF treatment, noting the significant impact of CFTR modulators in transforming the disease's prognosis.

The data from these trials will be presented at upcoming medical conferences, and the vanza triple could represent a major advancement in the treatment of cystic fibrosis, offering a new option for patients and potentially changing the course of the disease from its early stages.