News

The European Commission (EC) has expanded the approval of Kalydeco (ivacaftor) for the treatment of infants with cystic fibrosis (CF) as young as 1 month with at least one CFTR gating mutation. The approval makes Kalydeco, marketed by Vertex Pharmaceuticals, the first and only CFTR modulator…

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,…

Treatment with Kaftrio (elexacaftor, tezacaftor, and ivacaftor), sold as Trikafta in the U.S., may save people with cystic fibrosis (CF) an average of 15 minutes each day of respiratory physiotherapy to help with breathing, a study has found. The study, “Perceived burden of respiratory physiotherapy in people with…

A test of physical function called Glittre-ADL may be better than the standard six-minute walk test at identifying physical limitations in people with cystic fibrosis (CF), a study reports. “To our knowledge, this is the first study to evaluate the performance of CF patients on TGlittre and to compare…

In a recent study, scientists described the process through which they discovered GLPG2737, an investigational CFTR modulator considered by Galapagos and AbbVie to be a possible treatment for cystic fibrosis (CF). The molecule was found in early work to have an additive effect when combined with other types…

Treatment with Trikafta (ivacaftor/tezacaftor/elexacaftor) eases nose and sinus symptoms in eligible patients with cystic fibrosis (CF), and the improvement in nasal symptoms is accompanied by an increase in nasal nitric oxide (nNO), a biomarker. The findings suggest “nNO could be used as an objective, noninvasive outcome measure for…

A five-year study led by scientists at Emory University in Georgia aims to identify new ways to improve quality of life for people with cystic fibrosis (CF). The study, which will be led by Dio Kavalieratos, PhD, director of research at the Emory Palliative Care Center, is funded by…

A novel treatment strategy for cystic fibrosis (CF) that’s based on a cellular structure called the porosome showed promise in cell and animal models of CF caused by the common F508del mutation. In the cell model, this approach was more potent than tezacaftor and ivacaftor, which are CFTR…

Highly effective CFTR modulators, which target the cause of cystic fibrosis (CF), may ease signs of low iron levels in some patients with the disease, but low iron levels may remain even with treatment with the medications, a study finds. The scientists also emphasized that people with CF who…

Biopharma and biotech companies have launched more than 450 clinical trials into potential new treatments for cystic fibrosis (CF) since 2018, according to a report from Novotech, a contract research organization that specializes in helping drug developers to run trials. “There are ample opportunities presented by advancements in…