New preclinical data for TAVT-135, an experimental medication for the pan-genotypic treatment of cystic fibrosis (CF), support its effectiveness and utility in the treatment of patients who are ineligible for or do not respond well to cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Tavanta Therapeutics announced. The company shared the new data at both the…
A freeze-dried formulation of ivacaftor, ciprofloxacin, and L-salbutamol may be a novel approach to treating Pseudomonas aeruginosa infections and bronchoconstriction in patients with cystic fibrosis (CF), according to a new study published in DARU Journal of Pharmaceutical Sciences. The researchers prepared the microparticles with bovine serum albumin and L-leucine using the freeze-drying approach. After optimizing the process…
Patient screening in the ongoing phase 2 SPAN clinical trial researching a new enteric microgranulation formulation of adrulipase for the treatment of exocrine pancreatic insufficiency in patients with cystic fibrosis (CF) has been completed, First Wave BioPharma announced. “We plan to announce the completion of patient enrollment shortly and remain on track to report topline…
The US Food and Drug Administration (FDA) has approved ivacaftor (Kalydeco®) for the treatment of cystic fibrosis (CF) in pediatric patients ages 1 month to less than 4 months old who have at least 1 mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data. “Treating the…
The US Food and Drug Administration (FDA) has approved elexacaftor/tezacaftor/ivacaftor and ivacaftor (Trikafta®) for use in patients with cystic fibrosis (CF) aged 2 to 5 years who have at least 1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that might respond to elexacaftor/tezacaftor/ivacaftor and…
Among patients with cystic fibrosis (CF), rehabilitation is prescribed both before and after lung transplantation in an effort to optimize fitness and expedite post-transplant recovery, according to findings from a study published in Chronic Respiratory Disease. Evidence supports the use of exercise to improve pulmonary function, enhance health-related quality of life, and facilitate mucociliary clearance…
Researchers are developing a protocol that can be used to evaluate interventional trials on the management of pulmonary exacerbations of cystic fibrosis, according to details of a study published in Trials. Pulmonary exacerbations are a hallmark of cystic fibrosis. Around 25% of patients do not fully recover after experiencing one such episode; hence, they are…
A new study has found that the lymphocyte toxicity assay (LTA) may be a useful diagnosis and management tool for drug hypersensitivity reactions (DHRs) in patients with cystic fibrosis (CF). The study, published in Molecular Diagnosis & Therapy, also found evidence that toxic reactive metabolite accumulation might be part of the event cascade leading to…
In patients with cystic fibrosis (CF), the use of a novel vitamin D protocol has been associated with a rise in vitamin D levels and a reduction in the proportion of individuals with vitamin D insufficiency, according to findings from a multicenter, prospective, nonrandomized, uncontrolled experimental study published in Anales de Pediatría. Recognizing that vitamin…
In children 2 years of age or older with cystic fibrosis (CF), treatment with lumacaftor/ivacaftor (LUM/IVA) early in the disease course may modify disease progression, according to findings from a phase 2, placebo-controlled trial published in Annals of the American Thoracic Society. Among individuals with CF, mutations in the gene that encodes the CF transmembrane conductance…
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