News

Health Canada has extended its approval of Kalydeco (ivacaftor) to treat children as young as 2 months and weighing at least three kilograms, or about 6.6 pounds, who have cystic fibrosis (CF) caused by mutations that are responsive to the medicine. The extension covers patients who carry at…

Epithelial cells — the protective cells that line the airways — taken from people with cystic fibrosis (CF) exhibited distinct cellular and genetic features in lab cultures depending on whether they were obtained from the upper or lower airways, according to recent research. Those differences corresponded to distinct fluid-secreting…

BX004, an inhaled phage therapy being developed for chronic Pseudomonas aeruginosa lung infections, led to clinically meaningful improvements in lung function for cystic fibrosis (CF) patients with existing lung impairments, according to top-line results from the Phase 2 part of an ongoing Phase 1b/2a clinical trial. The therapy…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ARCT-032, Arcturus Therapeutics’ inhaled experimental treatment for cystic fibrosis (CF). The designation is intended to encourage the development of therapies for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides incentives,…

The European Commission has expanded the approval of Kaftrio (ivacaftor, tezacaftor, and elexacaftor) in combination with Kalydeco (ivacaftor) to include children ages 2 to 5 with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene. Previously cleared to treat patients ages 6 and older…

Cystic fibrosis (CF) patients who are positive for the fungus Aspergillus and develop an allergic reaction to it experience worse outcomes after getting COVID-19, a study reports. The allergic response, called bronchopulmonary aspergillosis (ABPA), increases the risk of hospitalization and sepsis, a life-threatening immune system reaction to an infection, data show.

Kaftrio (elexacaftor/tezacaftor/ivacaftor) — known as Trikafta in the U.S. — brings broad benefits in the form of better lung function, quality of life, and cognitive processing, while also helping to ease depression and improve stool frequency for cystic fibrosis (CF) patients, a real-world study in Italy found. For…

Treatment with CFTR modulators lessened the need for opioids and other painkillers in people with cystic fibrosis (CF), and led to a more stable use of psychotropics among those being treated for their mental health, a real-world data study from Australia reported. These therapies also led to increases…

While adding azithromycin to another antibiotic, called tobramycin, showed benefits in children with cystic fibrosis (CF) and early Pseudomonas aeruginosa infections, these outcomes were not due to the medication’s effects on the community of microbes  — known as the microbiome — living in the airways. That’s according to a new…

Loss of bladder or bowel control is common among adults with cystic fibrosis (CF), and such incontinence problems may impair patients’ quality of life and respiratory (breathing) care, regardless of the severity of their lung symptoms, a study found. “Persistent coughing” — a common symptom in cystic fibrosis —…