José Lopes, PhD, managing science editor —

The World Health Organization (WHO) approved ensifentrine as the recommended name to identify the cystic fibrosis (CF) treatment candidate RPL554. Verona Pharma’s ensifentrine is an inhaled inhibitor of the enzymes phosphodiesterase 3 and 4, designed to have bronchodilator and anti-inflammatory properties. The “-fentrine” staple indicates WHO’s…

Health Canada has approved Orkambi (ivacaftor/lumacaftor) to treat preschoolers with cystic fibrosis (CF) who carry the F508del mutation in both gene copies of the CFTR gene. Orkambi, developed by Vertex Pharmaceuticals, in the first disease-modifying therapy in Canada for children ages 2 to 5 with CF. It previously was …

Preschoolers with cystic fibrosis (CF) treated with inhaled hypertonic saline in a clinical trial showed significant improvement in lung function, PARI, the company whose solution and equipment was used in the study, reports. The Saline Hypertonic in Preschoolers (SHIP) study (NCT02378467) examined the effectiveness of treatment with 7% hypertonic saline…

Treatment with a triple combination — the new CFTR corrector VX-659, together with tezacaftor and Kalydeco (ivacaftor) — significantly improved lung function and was well-tolerated by cystic fibrosis (CF) patients, ages 12 and older, with at least one F508del mutation in two Phase 3 studies, according to Vertex Pharmaceuticals. This…

A protein toxin called Tse7 is used by Pseudomonas aeruginosa to kill neighboring bacteria, and may help explain why lung infections in cystic fibrosis patients are often dominated by a single P. aeruginosa strain, a new study reports. The research, “The Pseudomonas aeruginosa T6SS-VgrG1b spike is topped by…

Antibacterial Avycaz (ceftazidime-avibactam) showed significant in vitro efficacy against multidrug-resistant Pseudomonas aeruginosa collected from the sputum of cystic fibrosis (CF) patients, according to a recent study. The study, “Multidrug-resistant Pseudomonas aeruginosa from sputum of patients with cystic fibrosis demonstrates a high rate of susceptibility to ceftazidime–avibactam,” was…

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.

Blood levels of D-lactate may be markers of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF), according to preliminary data from a new study. The research, “Serum Level of D-Lactate in Patients with Cystic Fibrosis: Preliminary Data,” was published in the journal…

Orkambi (lumacaftor/ivacaftor), a treatment for cystic fibrosis (CF) patients in Australia age 6 and up, is closer to being subsidized by the government, according to Vertex Pharmaceuticals, the medication’s manufacturer. The company received a recommendation from the Pharmaceutical Benefits Advisory Committee (PBAC), an independent expert body, to list…

Arcturus Therapeutics’ RNA-based approach enables the production of healthy CFTR — the protein whose dysfunction is key in cystic fibrosis (CF) — and can be effectively delivered into lung cells, according to the company’s preclinical results. This CF treatment candidate is being developed in collaboration with Cystic Fibrosis…