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…
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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…
Among children with cystic fibrosis (CF), starting the approved oral therapy Orkambi (ivacaftor/lumacaftor) led to favorable changes in lung function and structure after one year, a real-world study shows. Improvements were seen in markers of small airway function and structural changes, including the lung clearance index (LCI), indicating…
Among cystic fibrosis (CF) patients treated with CFTR modulators in pivotal clinical trials, those who achieved lower sweat chloride levels — a standard assessment for diagnosing and monitoring CF — saw greater clinical improvements, according to a pooled analysis of trial data. Sweat chloride levels in patients also…
In cystic fibrosis (CF), certain immune cells show signs of dysfunction very early in life, according to a new study that used an animal model as well as blood samples from young children with CF. Researchers think immune cell deficiencies may influence the cycles of infection and inflammation that…
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