News

The U.K. Medicines and Healthcare Products Regulatory Agency approved Alyftrek (vanzacaftor, tezacaftor, and deutivacaftor) as a cystic fibrosis (CF) treatment for patients ages 6 and older. The triple-combination CFTR modulator, developed by Vertex Pharmaceuticals, is indicated for patients who have at least one copy of a responsive…

Kaftrio, a combination of elexacaftor, tezacaftor, and ivacaftor that’s marketed as Trikafta in the U.S., may ease breathing and digestive symptoms in adults with cystic fibrosis (CF) who’ve had a lung transplant, a study from the Netherlands suggests. Researchers did observe a slight, but significant, increase in creatinine,…

Biomx has raised $12 million in financing to advance BX004, a phage therapy being developed to treat bacterial infections — specifically, ones caused by Pseudomonas aeruginosa, known as P. aeruginosa for short — in people with cystic fibrosis (CF). The funding comes from investors purchasing the company’s stock,…

A European Medicines Agency (EMA) committee recommended that the label for Kaftrio be expanded to cover treatment for people with cystic fibrosis (CF) caused by a wider array of mutations, Kaftrio’s developer Vertex Pharmaceuticals said. The Committee for Medicinal Products for Human Use (CHMP) opinion will be reviewed…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Porosome Therapeutics’ new treatment strategy for cystic fibrosis (CF), which is based on the porosome, a type of cellular structure. The designation is given to boost the development of therapies for rare diseases, which affect…

Starting treatment with the CFTR modulator Orkambi (lumacaftor/ivacaftor) early in life may provide long-term benefits for children with cystic fibrosis (CF), yet lung function improvements may be temporary, according to a real-world study from Hungary. The study, “Body composition changes and clinical outcomes in pediatric cystic fibrosis…

Boehringer Ingelheim has launched a Phase 1/2 clinical trial of BI 3720931, an inhaled gene therapy designed to treat cystic fibrosis (CF). The first-in-human trial, dubbed LENTICLAIR 1 (NCT06515002), is expected to enroll about 36 adults with CF who are not eligible for treatment with CFTR…

Nearly 80% of men with cystic fibrosis (CF) who underwent fertility treatment due to a lack of sperm became biological parents, according to the largest study of its kind. “Assisted reproductive technologies have removed many limitations to parenthood for CF males,” researchers wrote in the study “…

Treatment with melatonin, a hormone best known for its sleep-promoting role, was found to normalize inflammatory responses to airway infection and reduce the burden of infection-causing bacteria in a mouse model of cystic fibrosis (CF), per new research by U.S. scientists. “Long-term systemic treatment with melatonin is an effective…

Young children with cystic fibrosis (CF) have less mature gut bacterial growth over time than do their typically developing peers, according to a new U.S. study. “Our findings demonstrate that the gut microbiota of infants with CF fails to undergo typical developmental maturation, instead remaining entrenched in a transitional-like…