News

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 —…

ViaNautis has secured $25 million in financing to advance work with polyNaut, its delivery platform for genetic and other treatments. The platform is designed to deliver a range of therapies into difficult-to-penetrate tissues. According to the company, the money raised will help to support polyNaut’s use…

A newborn screening program in New Jersey failed to identify cystic fibrosis (CF) in five children, all of whom went on to have severe lung disease, a new study reports. Based on the findings, New Jersey’s newborn screening program has been updated to include screening for many more disease-causing…

An MRI technique revealed signs of reversible bronchiectasis (widened and inflamed airways) in more than one in three people with cystic fibrosis (CF) being treated with Kaftrio, sold as Trikafta in the U.S., as part of a small one-year study in France. The technique, called ultrashort echo-time MRI, is…