Vertex Pharmaceuticals announced 10 scientific abstracts from the company's portfolio of cystic fibrosis, or CF, medicines are being presented at the 41st European Cystic Fibrosis Conference taking place June 6-9, 2018, in Belgrade, Serbia. Collectively, the data support 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 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 of KALYDECO 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 KALYDECO. Together, these studies provide further support for the benefit of both early and long-term treatment with CFTR modulators. Results from an interim analysis of the ongoing, 96-week EXTEND Phase 3 rollover study of tezacaftor/ivacaftor combination, approved in the U.S. as SYMDEKO 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. 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.
