Patricia Inácio, PhD, science writer —

New data suggest that inhalation of nitric oxide (NO) may improve lung function and alleviate the burden of serious lung infections, such as Mycobacterium abscessus complex (MABSC) and Pseudomonas aeruginosa, two serious pathogens affecting cystic fibrosis (CF) patients. Results from the Advanced Inhalation Therapies (AIT) pilot study were shared recently at the…

Celtaxsys has completed its Phase 2b trial assessing the safety and effectiveness of acebilustat (formerly CTX-4430) for cystic fibrosis (CF), with the company announcing the last visit of the last enrolled patient. Details of the trial, supported by the Cystic Fibrosis Foundation, will be presented at the upcoming 41st…

Patients with cystic fibrosis (CF) are at higher-than-average risk of developing gastrointestinal cancers, especially those who underwent a lung transplant, a new study shows. Transplant patients with CF were found in this retrospective study to have a five-times greater risk of gastrointestinal cancer than those who have not had a…

The Cystic Fibrosis Foundation will award up to $11 million to Icagen to support a multi-year research-program aiming to develop new therapies for patients with cystic fibrosis (CF) caused by nonsense mutations, who may fail to respond to the current CFTR modulator therapies. The aim of the research is to…

The two components of Orkambi act separately to trigger a liver enzyme that decreases the therapy’s ability to fight cystic fibrosis, a study reported. Vertex Pharmaceuticals created Orkambi by combining ivacaftor and lumacaftor (VX-809). It addresses the underlying cause of the disease — a gene mutation that leads to a build-up of…

Bacteria can transmit memory across generations so their descendants will know how to attach themselves to surfaces and form communities called biofilms, a study reports. Researchers’ findings shed light on cystic fibrosis-related bacteria’s early steps in biofilm formation. These pathogens cause persistent airway infections in people with the disease. The…

Ibuprofen does a good job of fighting bacteria in cystic fibrosis (CF) patients’ lungs, including two key threats, Pseudomonas aeruginosa and Burkholderia, a study shows. Researchers said the findings support ibuprofen’s use as an add-on therapy against bacterial infections in CF. The study, “Antimicrobial Activity of Ibuprofen against Cystic Fibrosis-Associated Gram-Negative…

Vertex Pharmaceuticals launched the first Phase 3 clinical trial investigating the triple combination therapy of VX-659, tezacaftor (VX-661), and Kalydeco (ivacaftor) in cystic fibrosis (CF) patients with one F508del mutation and one minimal function mutation in the CFTR gene, which is defective in CF patients. Vertex hopes the results…

AzurRx BioPharma announced that its clinical trial testing MS1819-SD as a replacement enzyme therapy for exocrine pancreatic insufficiency (EPI) associated with chronic pancreatitis (inflammation of the pancreas) has enrolled three new patients, completing 75 percent of its goal. Although patients with cystic fibrosis (CF) are excluded from the trial,…

Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) approved Symdeko (tezacaftor/ivacaftor and ivacaftor) as a therapy for cystic fibrosis (CF) patients carrying two copies of the F508del mutation in the CFTR gene (the gene defective in CF) or with one mutation that responds to tezacaftor/ivacaftor. The therapy was approved for…