News

A European Medicines Agency (EMA) committee has recommended expanding Kaftrio’s (ivacaftor, tezacaftor, and elexacaftor) label to be used in combination with Kalydeco (ivacaftor) in children with cystic fibrosis (CF) ages 2 to 5 who have at least one F508del mutation in the CFTR gene. The CFTR modulator…

Infections with Staphylococcus aureus small colony variants, known as SCVs, are highly prevalent in people with cystic fibrosis (CF) — and linked to the previous use of the broad-spectrum antibiotic trimethoprim sulfamethoxazole, a systematic literature review has found. SCVs are slow-growing bacteria that are associated with higher rates of chronic…

BX004, a virus-based cocktail designed to treat Pseudomonas aeruginosa infections in people with cystic fibrosis (CF), can reduce the number of bacteria in the lungs with no signs of resistance to treatment over time, a study has found. Findings from the first part of a small Phase 1b/2a…

A year of treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) significantly reduces recurrent nose and sinus inflammation — called chronic rhinosinusitis, or CRS — in people with cystic fibrosis (CF), though most patients still exhibit signs of severe sinus disease. These are the findings of a new U.S. study that, similar to…

A type of bacteria called Achromobacter, which can cause problematic lung infections in people with cystic fibrosis (CF), uses a molecular weapon — one known as a type 3 secretion system — to induce a powerful inflammatory response during infections, a new study found. “These bacteria resist the action…

LasB, an enzyme that promotes Pseudomonas aeruginosa infections in the lungs of people with cystic fibrosis (CF), is significantly more active in the early stages of infection with the bacteria, known more simply as P. aeruginosa, a study revealed. These findings suggest that therapies targeting LasB may be…

CFTR — the protein whose defect or absence causes cystic fibrosis (CF) — helps cells called ionocytes remove liquid from the airways, a study shows. The finding sheds new light on the biological processes that are disrupted in CF and may have important implications for treating the disease. The…

SPL84, an experimental treatment being developed for people with cystic fibrosis (CF) that’s caused by a specific mutation, showed a good safety profile in a first clinical trial in healthy volunteers, according to its developer, SpliSense. Spurred by these positive results, SpliSense now is planning a Phase 2 clinical…

A newly identified therapeutic target showed a potential to enhance the effectiveness of antibiotics against resistant Mycobacterium abscessus (Mabs) lung infection, a damaging complication in people with cystic fibrosis (CF), researchers report. Experiments using human airway organoids derived from CF patients also accurately reflected many features seen in their…

Adults using Kalydeco (ivacaftor) or Trikafta (ivacaftor/tezacaftor/elexacaftor) to treat their cystic fibrosis (CF) showed gains in lung function and nutritional status, according to a real-world, observational study in Australia. In contrast, no significant improvements in lung function were seen in adults or children with CF using Orkambi (ivacaftor/lumacaftor)…