Andrea Lobo, PhD, science writer —

The U.S. Food and Drug Administration (FDA) has granted fast track designation to SPL84, an experimental inhalation therapy for people with cystic fibrosis (CF) caused by the 3849+10 kb C-to-T mutation in the CFTR gene. The designation is given to accelerate the development and review of therapies meant to address unmet…

Improvements in mucus clearance with the approved medication Trikafta — evident among patients within a month after starting treatment, according to researchers — may contribute to better lung function in people with cystic fibrosis (CF), per a new U.S. study. Mucus clearance mechanisms are meant to keep the…

Four years after the approval of Trikafta (elexacaftor/tezacaftor/ivacaftor) for cystic fibrosis (CF), global disparities in access to the treatment remain and mainly affect low- and middle-income countries, according to a recent study. “The extent of international disparities, in combination with the high price, suggested that without urgent action,…

A history of pancreatic inflammation, diabetes, high blood pressure, and high cholesterol may increase the risk of pancreatic cancer in people with cystic fibrosis (CF), according to a large database report. These patients also had a higher prevalence of known risk factors for pancreatic cancer, including smoking, alcohol consumption,…

People with advanced liver disease associated with cystic fibrosis (CF) have more pro-inflammatory bacteria in their feces relative to those without CF- associated liver disease, a study shows. The findings suggest similar changes in the gut microbiota, or the populations of friendly microbes that naturally live in the intestines. This…

A greater increase in body mass index (BMI), a measure of body fat, from ages 1 through 6 in boys with cystic fibrosis (CF) results in the early production of sex hormones, called adrenarche, according to a small study. Boys with early adrenarche had…

The nasal microbiome — the community of microorganisms that live in the nose — obtained through nasal lavage correlates with inflammation in the nose and sinuses of children with cystic fibrosis (CF),  a study indicates. The effect wasn’t observed when samples were obtained by a nasal…

Clarametyx Biosciences has completed a $33 million financing round to support its development of CMTX-101, a non-antibiotic treatment candidate for persistent bacterial infections in people with cystic fibrosis (CF). The funding contributed to the start of a Phase 1b/2a clinical trial of CMTX-101 as an adjunct therapy to standard-of-care…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to BX004, BiomX’s inhaled phage therapy being developed for chronic Pseudomonas aeruginosa pulmonary infections in people with cystic fibrosis (CF). The designation seeks to encourage new therapies for rare diseases, those affecting fewer than 200,000…

Treatment with Kaftrio (elexacaftor/tezacaftor/ivacaftor) improved the pancreatic function of two women with cystic fibrosis (CF), allowing them to stop their pancreatic enzyme replacement therapy (PERT). The women each had a combination of an F508del mutation, which leads to the abnormal folding of the CFTR protein, and…