News

A case report about a 10-year-old boy suggests that Vertex Pharmaceuticals‘ Kalydeco (ivacaftor) can restore the function of the pancreas and the production of pancreatic enzymes in children with cystic fibrosis (CF), leading to a reduction or discontinuation of pancreatic enzyme replacement therapy (PERT). The…

Data from Phase 1 studies in healthy people and preclinical work indicate that ELX-02 may help to treat cystic fibrosis (CF), Eloxx Pharmaceuticals, its developer, reported. The therapy was well-tolerated in single- and multiple-ascending dose studies in those clinical trials, and raised levels of the crucial CFTR protein in…

Claire’s Place Foundation is throwing an inaugural fundraising ball June 29 to celebrate the life and legacy of the foundation’s late founder, Claire Wineland, who had cystic fibrosis (CF). Called the Clairity Ball, the event will be held at the Fairmont Miramar Hotel & Bungalows in Santa Monica,…

Proteostasis Therapeutics received orphan drug designation from the European Commission for its investigational CFTR amplifier PTI-428 in development for the treatment of cystic fibrosis (CF). This recognition of the therapeutic potential of PTI-428 in Europe follows previously granted orphan drug, breakthrough therapy, and fast…

Manuka honey, produced from the nectar of a flowering plant native to Australia and New Zealand, showed significant anti-Pseudomonas aeruginosa activity in an experimental model of lung infection designed to closely replicate the pulmonary environment of patients with cystic fibrosis (CF). The results were reported in the study “Anti-pseudomonad…

Armata Pharmaceuticals‘ investigational phage therapy AP-PA01 combined with antibiotics holds promise for treating multidrug-resistant Pseudomonas airway infections, updated data from a case study show. A young woman with cystic fibrosis (CF) was successfully treated for a multidrug-resistant Pseudomonas aeruginosa pneumonia using AP-PA01 and antibiotics. At 100 days…

Vertex Pharmaceuticals selected its VX-445 (elexacaftor) triple regimen to apply for regulatory approvals in the U.S. and Europe over the third and fourth quarters of 2019. The therapeutic regimen is intended for the treatment of cystic fibrosis…

Inhalation of nitric oxide as an add-on therapy may help reduce the burden of serious bacterial lung infections, namely the hard-to-treat Mycobacterium abscessus, in cystic fibrosis (CF) patients, AIT Therapeutics announced. The study with that finding, “Pilot study to test inhaled nitric oxide in cystic fibrosis…

International Biophysics, the maker of AffloVest, announced that it is continuing to support people with cystic fibrosis and efforts to raise awareness about this disease, donating 50 of its airway clearance therapy vests to be distributed across the U.S. AffloVests are being given away, one in each state,…

Treatment with ABBV-2222, an investigational CFTR corrector formerly known as GLPG2222, was well-tolerated and partially corrected the function of the CFTR protein, both alone and in combination with Kalydeco (ivacaftor), in a group of patients with cystic fibrosis (CF), Phase 2 clinical trials show. Trial findings…