José Lopes, PhD, managing science editor —

The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to Proteostasis Therapeutics’ cystic fibrosis therapy PTI-428 — a move aimed at accelerating its development. Mutations of the CFTR gene cause CF. The most common is F508del, which leads to faulty CFTR protein. Normal CFTR protein is required for transporting chloride ions…

Pulmonary hypertension increases the risk of cystic fibrosis patients’ lung function worsening, but is not a predictor of reduced survival, a study reports. The research, ”Prognostic significance of pulmonary hypertension in patients with cystic fibrosis: A systematic review and meta-analysis,” was published in the journal Medicine.

The U.K.’s Medicines & Healthcare products Regulatory Agency (MHRA) issued an alert to urge patients and healthcare professionals to stop using Aquilon‘s medical nebulizers. Among those affected are patients with cystic fibrosis and asthma. All medical devices marketed in Europe need to have a mandatory CE certification,…

A Phase 1 clinical trial testing Arch Biopartners’ AB569 for the treatment of antibiotic-resistant bacterial infections is now enrolling healthy volunteers. The therapy candidate could be a new help for patients with cystic fibrosis (CF), chronic obstructive pulmonary disease (COPD), and other respiratory conditions. The trial will evaluate the…

The U.S. Food and Drug Administration (FDA) gave a green light to human testing of ORC-13661,  Oricula Therapeutics‘ treatment candidate to prevent hearing loss in people taking high doses of aminoglycoside antibiotics, including patients with cystic fibrosis. Approval given the company’s Investigational New Drug Application will enable Oricula to test…

The interaction of ions and sugars called glycans can affect the mucus that lines airways — a finding that has implications for cystic fibrosis, two studies reported. CF is characterized by abnormally thick and sticky mucus that can damage the lungs, gut and other organs and breed bacteria. The two studies showed…

More than half of British babies born with cystic fibrosis today will reach the age of 50, according to a new forecasting method. The same is true of CF patients who have reached the age of 30 today, researchers said. Reaching 30 is a milestone that signals another 20 years of…

The most influential cystic fibrosis advocacy groups in the United States and Europe have endorsed the idea of a Phase 1 clinical trial to test a triple-combo therapy that Proteostasis Therapeutics has developed, the company announced. Proteostatis said the endorsements came from the Cystic Fibrosis Foundation’s and European CF Society’s clinical…

Multi-drug-resistant nontuberculous mycobacteria (NTM) can be found in the gastric juice and tubes that are used to feed some cystic fibrosis (CF) patients, research shows. Tube feeding, known as gastrostomy, involves surgeons inserting a tube through the abdominal wall and into the stomach. Gastrostomy can also be used to drain…

The activity of Zerbaxa (ceftolozane/tazobactam, or C/T) alone or in combination with other antibiotics shows promise against multi-drug resistant (MDR) Pseudomonas aeruginosa infection in pediatric cystic fibrosis (CF) patients, new research shows. The study, “In vitro Activity of Ceftolozane/Tazobactam Alone or with an Aminoglycoside Against Multi-Drug-Resistant Pseudomonas aeruginosa…