News

The European Commission approved expanding the use of Orkambi (lumacaftor/ivacaftor) to include children as young as 1 year old with cystic fibrosis (CF) and two copies of the F508del mutation in the CFTR gene. Vertex Pharmaceuticals’ therapy was first approved in the European Union…

The first patient has been dosed in a Phase 1 clinical trial of KB407, Krystal Biotech’s investigational gene therapy for people with cystic fibrosis (CF) regardless of the type of disease-causing CFTR gene mutation. The Phase 1 CORAL-1/US study (NCT05504837) is recruiting up to 20 adults with…

The presence of intestinal metabolites called bile acids in the airways of 1-year-old children genetically diagnosed with cystic fibrosis (CF) is significantly associated with early disease mechanisms and worse clinical outcomes, according to a study in Australia. These include increased levels of airway inflammation biomarkers, greater structural lung damage,…

Treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) can improve lung function and nutritional status for people with cystic fibrosis (CF) caused by the N1303K mutation, a small real-world study reports. The N1303K mutation is not among the 178 disease-causing mutations in the CFTR gene considered to be responsive to Trikafta and…

First Wave BioPharma has finished dosing in its Phase 2 clinical study, SPAN, in which a new adrulipase formulation is being tested as a potential exocrine pancreatic insufficiency (EPI) treatment in people with cystic fibrosis (CF). With the last patient dosed, the company remains on track for next…

Researchers have synthesized two new molecules that are able to kill Staphylococcus aureus and Pseudomonas aeruginosa — two species of bacteria that commonly cause lung infections in people with cystic fibrosis (CF). These new molecules, known as synthetic nano-engineered antimicrobial polymers or SNAPs, showed synergistic bacteria-killing effects when combined with…

Dosing has begun in a Phase 1 clinical trial of CM001, an inhaled treatment designed to act as a molecular prosthetic in cells and treat cystic fibrosis (CF) regardless of a patient’s underlying mutation, Cystetic Medicines, the therapy’s developer, announced. Most immediately, the company expects that CM001…

Trikafta treatment for six months in people with cystic fibrosis (CF) was found in a real-world study to reduce the thickness of the airway walls and lead to less excessive mucus plugging in the lungs. Overall, as observed by MRI, the approved CF therapy improved structural abnormalities in…

Kaftrio improved lung function for up to 2.5 years in cystic fibrosis (CF) patients with advanced disease who had early access to the medication in Spain, according to a real-world study. Taking Kaftrio, a triple combination of elexacaftor, tezacaftor, and ivacaftor, also reduced the frequency of exacerbations, when…

In the months after starting on Trikafta, people with cystic fibrosis (CF) generally experience lower rates of hospitalizations and lung infections — but nonetheless, the total cost of healthcare for these patients tends to increase, a new economic analysis indicates. The findings are in line with prior analyses…