Vanza Triple Success in Cystic Fibrosis Phase III

Vertex Pharmaceuticals has announced encouraging outcomes from its Phase III trials for a new cystic fibrosis (CF) treatment known as vanza triple. The company's late-stage trials, which include SKYLINE 102, SKYLINE 103, and RIDGELINE 105, evaluated the efficacy and safety of vanza triple in CF patients of different age groups.

The SKYLINE trials, involving participants aged 12 and above, compared vanza triple to Vertex’s existing CF medication, Trikafta. The new treatment, which contains vanzacaftor, tezacaftor, and deutivacaftor, demonstrated non-inferiority to Trikafta in improving lung function, measured by forced expiratory volume in one second (FEV1), over a period of 24 weeks. Furthermore, vanza triple was found to be more effective in reducing sweat chloride levels, a significant indicator in CF treatment, and maintained this effect for up to 52 weeks.

The RIDGELINE 105 study, which targeted younger patients between six and 11 years old, corroborated the SKYLINE findings. It showed a significant reduction in mean sweat chloride levels and a high percentage of children achieving sweat chloride concentrations below the CF diagnostic threshold.

Safety data from all three trials indicated that vanza triple was well-tolerated, with adverse events generally mild to moderate and in line with CF symptoms. Serious adverse events were rare and evenly distributed between the treatment groups.

Carmen Bozic, Vertex’s Chief Medical Officer, expressed satisfaction with the results, noting that vanza triple has the potential to surpass the high standards set by Trikafta. The company is planning to submit a New Drug Application in mid-2024, utilizing a priority review voucher to expedite the review process to six months.

Vertex also intends to apply for a Marketing Authorization with the European Medicines Agency. The positive results from the Phase III trials add to Vertex’s recent successes, including FDA approvals for its gene-edited cell therapy and a non-opioid pain medication that has shown significant pain relief in various clinical trials.

The development of vanza triple represents a significant step forward in the treatment of cystic fibrosis, offering a promising alternative to existing therapies and potentially improving the quality of life for many patients.