Steve Bryson, PhD, science writer —

Kaftrio successfully improved lung function and quality of life in three Italian cystic fibrosis (CF) patients who have severe lung disease with an unknown mutation in the CFTR gene in addition to the common F508del mutation, a case series reported. Kaftrio (elexacaftor, tezacaftor, and ivacaftor), sold as Trikafta in the…

Vertex Pharmaceuticals is initiating two Phase 3 clinical trials into the safety and efficacy of a potentially new, next-generation triple combination therapy for cystic fibrosis (CF): VX-121 plus tezacaftor and VX-561 (deutivacaftor). Expected to begin later this year, the development program will compare this once-daily therapy with Vertex’s…

Most primary care providers (PCPs) in Ontario — family physicians, pediatricians, and midwives — voiced a willingness and confidence in providing some level of care to infants testing positive for cystic fibrosis (CF) in newborn screening programs, a Canadian survey reported. A majority favored treating minor illnesses like infections…

Inflammation in the airways of people with cystic fibrosis (CF) regulates the pH of the surface liquid and increases the response to approved CFTR modulators, a cell-based study suggested. Airway inflammation may be a key determinant in response to CF medicines, the researchers noted. “To advance personalized CFTR…

Vertex Pharmaceuticals has reached reimbursement agreements with health authorities in France and Italy for its various cystic fibrosis (CF) treatments, including Kaftrio (ivacaftor/tezacaftor/elexacaftor), a next-generation therapy approved for use with ivacaftor. With these agreements — which bring the treatments into each country’s public health program — about 3,000 CF patients…

Reduced levels of the VIP hormone in the pancreas due to low nerve supply may contribute to the development of early diabetes in people with cystic fibrosis (CF), a study in mice suggests. The study, “VIP reduction in the pancreas of F508del homozygous CF mice and early signs…

Two months of treatment with Kalydeco (ivacaftor) by cystic fibrosis (CF) patients who carry a CFTR gating mutation significantly increased the diversity of bacteria in the gut but not in the respiratory system, a small study reported. A…

Human mesenchymal stem cells (hMSCs) effectively treated infections caused by non-tuberculous mycobacteria in cell-based tests and a mouse model of cystic fibrosis (CF), a study revealed.  Although this strategy may help treat those infections in people with CF and other lung…

Antibiotic-resistant infection with Mycobacterium abscessus bacteria in people with cystic fibrosis (CF) can evolve rapidly to become more virulent, a genetic analysis suggests.  The findings support early testing and treatment of lung infections before symptoms emerge to…

Eloxx Pharmaceuticals has announced that it’s now added the approved cystic fibrosis (CF) therapy Kalydeco (ivacaftor) to its global Phase 2 clinical program for ELX-02. Ongoing ELX-02 Phase 2 clinical trials, launched in 2019 with four treatment…