Vertex Pharmaceuticals data presented at the 41st European Cystic Fibrosis Conference supports the potential disease-modifying benefits of treating the underlying cause of CF and Vertex’s progress toward enhancing and expanding treatment options for all people living with CF.

Data from the ongoing Phase 3, open-label ARRIVAL study presented at an oral session and published online today in The Lancet Respiratory Medicine show that treatment with Kalydeco (ivacaftor) resulted in substantial decreases in mean sweat chloride as well as improvements to multiple efficacy endpoints, suggesting the potential to preserve pancreatic function and modify the course of CF beginning in children as young as one year of age.

In addition, final annual analyses of the completed, five-year, post-approval observational safety study show that patients taking Kalydeco had lower risk of death, transplantation, hospitalization and pulmonary exacerbations compared to patients who were matched on age, gender and genotype class who did not receive the drug. Together, these studies provide further support for the benefit of both early and long-term treatment with CFTR modulators.

In addition, results from an interim analysis of the ongoing, 96-week EXTEND Phase 3 rollover study of Symdeko (tezacaftor/ivacaftor combination) also add to the growing body of evidence supporting the benefit of long-term treatment of the underlying cause of the disease. Analysis presented during a poster presentation shows that the initial improvements in lung function (measured by the absolute change in percent predicted forced expiratory volume in one second [ppFEV1]) observed in the Phase 3 EVOLVE study of patients homozygous for F508del were sustained for up to 48 weeks (24 weeks in EVOLVE + 24 weeks in EXTEND). Treatment was well-tolerated, demonstrating a safety profile consistent with that observed in the pivotal EVOLVE and EXPAND studies. Improvements across some secondary endpoints that were observed in the parent study were maintained in patients homozygous for F508del (n=459).

“The data presented at ECFS are further evidence that treating the cause of CF may significantly slow the progression of this disease beginning early in life, underscoring the importance of starting treatment for eligible patients as early as possible,” said Reshma Kewalramani, MD, Executive Vice President and Chief Medical Officer at Vertex. “Over the past year, we’ve made rapid progress in developing multiple new medicines that treat the underlying cause of CF, and today, we are closer to our goal of developing medicines for all patients with CF than ever before.”