News

The U.S. Food and Drug Administration gave positive feedback to the design of AmpliPhi Biosciences’ two proposed clinical trials of its Pseudomonas aeruginosa-targeting treatment candidate AB-PA01, the company announced in a press release. P. aeruginosa is the major cause of lung infections in cystic fibrosis patients.

A new electronic nose, called Aeonose, was seen to be a feasible, accurate and easy-to-use diagnostic test for cystic fibrosis (CF) in children and adolescents, a small pilot study shows. The research “Feasibility and diagnostic accuracy of an…

Blocking superoxide dismutase, the enzyme that responds to stress, in Pseudomonas aeruginosa bacteria improves antibiotics’ efficacy, a Canadian study reported. The study, “Superoxide dismutase activity confers (p)ppGpp-mediated antibiotic tolerance to stationary-phase Pseudomonas aeruginosa,” was published in Proceedings of the National Academy of Sciences (PNAS). “We identified a new function important…

Cystic fibrosis patients who failed to show early improvements in lung function while being treated with Orkambi (lumacaftor/ivacaftor) still experienced fewer pulmonary exacerbations than those given a placebo in Phase 3 studies, a pooled analysis of those trials reports. Clinicians should consider the totality of outcomes when examining the…

Mutations in the CFTR gene that associate with severe disease can also lead to cardiac problems — specifically, the development of subclinical left ventricular dysfunction — in cystic fibrosis patients, a study suggests. The study, “Subclinical Left Ventricular Dysfunction is Influenced by Genotype Severity in Patients with Cystic…

Vertex Pharmaceuticals has completed enrollment for two Phase 3 studies testing the triple combination therapy of its new CFTR corrector VX-659 together with tezacaftor (VX-661) and Kalydeco (ivacaftor) in patients with cystic fibrosis (CF). The studies focus on patients with two F508del mutations in the CFTR gene (the…

Editor’s Note: This is the second of a four-part series looking at Vertex Pharmaceuticals and its “all in for CF” approach. Here, we detail the steps that made three disease-modifying oral treatments for cystic fibrosis possible. Kalydeco (ivacaftor), as Vertex‘s cystic fibrosis potentiator came to be known, was a small…

Beginning Oct. 1, the cystic fibrosis (CF) targeted therapy Orkambi (lumacaftor/ivacaftor) will be included on Australia’s Pharmaceutical Benefits Scheme. The Australian government’s decision resulted from the completion of an agreement with Orkambi’s manufacturer, Vertex Pharmaceuticals, and was supported by the recent positive recommendation from the Pharmaceutical Benefits Advisory Committee…

A research team at The University of Queensland was honored for its work on the spread of bacteria among cystic fibrosis patients, being named a winner of this year’s Australian Museum Eureka Prizes. The prizes recognize excellence in four fields:  research and innovation, science engagement, science leadership, and science education. CF Air, as…

Vertex Pharmaceuticals and Genomics announced a three-year collaboration to better understand genetic variations and their effect on clinical outcomes of diseases with unmet therapeutic needs, including cystic fibrosis. The collaboration also will promote the development of precision medicine, an approach to customize treatment based on each patient’s genetic…