News

Pulmonary flares seen as key sex difference in patients on Kalydeco

Despite treatment with Kalydeco (ivacaftor), females with cystic fibrosis (CF) continue to experience more lung exacerbations — periods when CF respiratory symptoms suddenly worsen — than do males, for reasons that are not well understood, a large patient registry study reported. “Our findings demonstrate that sex disparities…

CF patients eat high-fat diet despite guidelines: Review

Despite recent guidelines recommending nutrient consumption similar to the general population, many adults and late adolescents with cystic fibrosis (CF) still follow a high-fat, high-energy diet, according to a review done by researchers in Australia. The findings suggest that “overall, diet intakes of adults with CF appear to be…

Phase 2 trial possible in ARCT-032, inhaled mRNA therapy for CF

Arcturus Therapeutics plans to soon submit an application to the U.S. Food and Drug Administration (FDA) seeking permission to begin testing multiple ascending doses of ARCT-032, an inhaled experimental treatment for cystic fibrosis (CF). The planned Phase 2 study, supported by promising safety and preliminary efficacy data…

FDA decision on vanza triple, new CFTR modulator, due by Jan. 2

Authorities in the U.S. and Europe are reviewing applications from Vertex Pharmaceuticals seeking approval of the company’s novel CFTR modulator triple combination, vanzacaftor, tezacaftor and deutivacaftor.  Vertex is asking that the vanza combo be approved for people with cystic fibrosis (CF) ages 6 and older who carry…

3 CFTR modulators guaranteed public reimbursement in England

Vertex Pharmaceuticals and England’s National Health Service (NHS) have reached an agreement that allows all current and future eligible cystic fibrosis (CF) patients to access three of the company’s approved CFTR modulators at low or no out-of-pocket cost. Kaftrio (elexacaftor/tezacaftor/ivacaftor, sold as Trikafta in the U.S.), Symkevi (tezacaftor/ivacaftor,…