The European Commission has granted orphan drug designation to ARCT-032, an inhaled experimental treatment for cystic fibrosis (CF) developed by Arcturus Therapeutics. Orphan drug designation is designed to encourage developing therapies for rare and serious diseases, those affecting not more than 5 in every 10,000 people in the European Union…
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A European Medicines Agency (EMA) committee recommends extending the label for Kalydeco (ivacaftor) to children with cystic fibrosis (CF) as young as a month who carry at least one CFTR gating mutation. If the European Commission (EC) accepts the endorsement by the Committee for Medicinal Products for Human…
Treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) significantly reduced rates of colonization with the bacterium Pseudomonas aeruginosa in adults with cystic fibrosis (CF) at a center in Kentucky. Trikafta also reduced the need for mucus-clearing medicines and antibiotics, according to the study “Impact of Elexacaftor/tezacaftor/ivacaftor on Respiratory Colonization…
ReCode Therapeutics has launched a Phase 1 clinical trial to evaluate the safety of RCT2100, its investigational genetic medicine for cystic fibrosis (CF), in healthy volunteers and the first group of participants has been dosed. RCT2100 is being developed for CF patients with class I mutations in the…
Compounds secreted by Lactobacillus, a group of bacteria commonly used in probiotics, are able to impede the growth of Pseudomonas aeruginosa isolated from the lungs of people with cystic fibrosis (CF). That’s according to the study, “Cell-free supernatants from Lactobacillus strains exert antibacterial, antibiofilm, and antivirulence…
In children with cystic fibrosis (CF) treated with Orkambi (ivacaftor/lumacaftor), the levels of the medication reached in the blood depend on how much the children weigh and how well their liver is working, a new study discovered. These findings offer insights into how to best adjust doses of the approved…
People with cystic fibrosis (CF) can have abnormal levels or metabolism of three “essential nutrients” — vitamin D, calcium, and phosphate — even when using pancreatic enzyme replacement therapy (PERT) and taking dietary supplements, a small study suggests. “The findings of this study have many practical implications in…
Intellia Therapeutics and ReCode Therapeutics are joining forces to develop new genomic medicines for people with cystic fibrosis (CF). The companies said initial research will focus on patients who have “limited or no treatment options available.” The ultimate goal is to create a new genomic medicine that…
Certain markers of inflammation found deep in the lungs of children with cystic fibrosis (CF) can help predict how bronchiectasis will worsen over time, offering insights for better treatment and care, a study suggests. Among other markers, higher levels of interleukin-8 (IL-8), a signaling…
Between 2011 and 2021, improving lung function and better survival rates were consistently seen among people with cystic fibrosis (CF) in Europe, particularly in wealthier countries and with the introduction of new treatments, according to a study that tracked more than 45,000 CF patients across the continent over the…
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