- New clinical data show TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) continues to deliver significant benefit across a variety of outcomes and over the long term in people with CF -

BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that multiple abstracts on the company’s portfolio of cystic fibrosis (CF) medicines will be presented in posters and oral presentations at this year’s North American Cystic Fibrosis Conference (NACFC), including studies demonstrating the clinical benefits and long-term safety of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor).

Vertex will present new long-term safety and efficacy data on TRIKAFTA from an open-label 192-week extension study in people 12 years and older with CF and at least one F508del allele. Based on an interim analysis through Week 144 of the 192-week study, people receiving TRIKAFTA showed maintained improvements in lung function, respiratory symptoms and cystic fibrosis transmembrane conductance regulator (CFTR) function. TRIKAFTA also continues to be generally well tolerated (Poster #170 and Oral Workshop: W21.2). These data will also be discussed during the second plenary on November 4, 2022, at NACFC.

“These long-term data at 144 weeks demonstrate remarkable health effects in the longest study of TRIKAFTA. The data show sustainment of the historic improvements in lung function, respiratory symptoms and sweat chloride, a marker of CFTR function,” said Deepika Polineni, M.D., MPH, Associate Professor of Pediatrics and Cystic Fibrosis Center Director at Washington University School of Medicine in St. Louis, and a co-investigator of the 445-105 study (Poster #170). “CFTR modulators like TRIKAFTA have pivotally changed standard of care therapy in CF, and the CF community continues to benefit from ongoing collection and evaluation of long-term data.”

Vertex will also present data for the first time from its investigational Phase 3 open-label study designed to evaluate the safety, pharmacokinetics and efficacy of TRIKAFTA in children 2 through 5 years of age with CF and at least one F508del allele (Poster #693). The study shows that treatment with TRIKAFTA led to improvements in sweat chloride concentration and lung function, as measured by the lung clearance index, and stable nutritional status in children 2 through 5 years of age. TRIKAFTA was generally well tolerated, with a safety profile generally consistent with older age groups. Based on these results, Vertex recently submitted a New Drug Application (NDA) with the U.S. Food and Drug Administration for this age range and will be filing for approvals with the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) by the end of this year.

Additionally, Vertex will present data from a pooled analysis from multiple Phase 3 studies with CFTR modulators evaluating how the restoration of CFTR-mediated chloride transport, as reflected by changes in sweat chloride concentration, impacts clinical outcomes in people with CF treated with CFTR modulators (Poster #694). The study showed that people with CF ages 12 years and older treated with CFTR modulators achieved higher levels of CFTR activity, as reflected in lower levels of sweat chloride. Those with greater improvements in sweat chloride demonstrated greater improvements in lung function, respiratory symptoms, body mass index, pulmonary exacerbations, and had better lung function trajectory over time. These new data demonstrate that higher levels of CFTR function as measured by a reduction of sweat chloride are associated with improved clinical outcomes. The best outcomes were seen in those achieving a sweat chloride concentration