Marisa Wexler, MS,  senior science writer—

ELX-02, an investigational treatment of cystic fibrosis due to nonsense mutations, has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). This designation is given to treatments with the potential to improve care for rare diseases, defined as those affecting fewer than 200,000 people in…

The Scottish Government and Vertex Pharmaceuticals have reached a deal to make Vertex’s Kaftrio (ivacaftor/tezacaftor/elexacaftor) available for people with cystic fibrosis (CF) once the medication receives its European license. This follows similar agreements in the three other U.K. countries, England, Wales, and Northern…

Levels of inflammatory markers in the lungs may help predict pulmonary exacerbations in children with cystic fibrosis (CF), a new study suggests. The study, “BAL inflammatory markers can predict pulmonary exacerbations in children with cystic fibrosis,” was published in the journal Chest. In people…

Trikafta (elexacaftor/tezacaftor/ivacaftor combo) improves lung function in cystic fibrosis (CF) patients who have one F508del mutation and either one gating mutation (F/G) or one residual function mutation (F/RF) in the CFTR gene, new clinical trial data show. That finding was announced by Vertex Pharmaceuticals, which markets Trikafta.

Measuring levels of a molecule called lipoarabinomannan, or LAM, in a person’s urine could be an easier way of screening for airway infections due to nontuberculous mycobacteria in people with cystic fibrosis (CF), a study suggests. The study, “Urine lipoarabinomannan as a marker for low-risk…

The number of adults with cystic fibrosis (CF) living in the U.K. is expected to increase by at least 28% in 10 years, and 36% of these adults will be older than 40, a study projects. Findings highlight the need to make plans now to care for this changing…

A five-year, $3.2 million grant from the National Institutes of Health (NIH) will fund a  research project that aims to better understand the interactions between bacteria and mucus. Results could help inform new ways of treating diseases where bacterial infections that involve mucus are problematic, such as cystic…

ARINA-1, a nebulized therapy being developed by Renovion for cystic fibrosis (CF) and other chronic inflammatory lung diseases, may be better than currently available options for clearing mucus in CF patients with the most common CFTR mutation, results of a study in cell models of CF showed. The…

Rage Biotech, a new company building on research done at leading Australian universities, aims to develop treatments to help people with chronic inflammatory lung diseases, including cystic fibrosis, severe asthma, and chronic obstructive pulmonary disease. Supported by investments from the IP Group, Monash University, and…

NHS England and Vertex Pharmaceuticals have reached an agreement that will allow access to Vertex’s Kaftrio (ivacaftor/tezacaftor/elexacaftor), a triple combination therapy for cystic fibrosis (CF) to be used in a combination regimen with Kalydeco, as soon as the medication is approved by the European…