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…
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…
Trikafta (elexacaftor/tezacaftor/ivacaftor) significantly lowered several pro-inflammatory blood markers with as early as one month of use in cystic fibrosis (CF) patients carrying F508del — the most common CF-causing mutation — and in parallel with gains in lung function. These are the findings of a small study that suggest…
Tezacaftor, a medication used to treat cystic fibrosis (CF), may unexpectedly trap certain fats in the lungs, diverting their use as chemical raw material that the body converts into ceramides, which are needed for the brain to develop and respond to stress, according to…
Among young children with cystic fibrosis (CF), starting the approved oral therapy Orkambi (ivacaftor/lumacaftor) led to favorable changes in nutrition and growth status during the first six months of treatment, according to a small real-world study. Orkambi, first approved in the U.S. in 2015 and now indicated for…
Danish children with cystic fibrosis (CF) born between 2000 and 2022 showed noticeable gains in growth during their first five years of life, with those born after 2016 seeing the most progress in reaching normal growth standards, a study shows. This is likely due to newborn screening programs (NBS)…
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,…
Children with cystic fibrosis (CF) who are being treated with Symdeko (ivacaftor/tezacaftor) or Orkambi (ivacaftor/lumacaftor) experience few to no symptoms of chronic rhinosinusitis and maintain a normal sense of smell, a study in Denmark found. Although small, the study suggests that not only Trikafta, a triple…
Despite some similarities, the lung composition of microbes and their antimicrobial resistance profiles differed between people with bronchiectasis and cystic fibrosis (CF) and those with non-CF bronchiectasis, a study found. The findings suggest “the need for customized management strategies for each disease,” the researchers wrote in the study, “…
CFTR modulators may help control glucose (sugar) levels in the blood of people with cystic fibrosis (CF), according to researchers in Israel, who called for regular oral glucose tolerance testing to adjust insulin dosing in diabetes and keep glucose from dropping too low. The study, “Long-Term therapy with…
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