Alice Melão, MSc,  —

Treatment with lenabasum can effectively prevent inflammation and induce pro-resolving signals in airway immune cells from patients with cystic fibrosis (CF), results from a preclinical study show. Lenabasum’s more recent preclinical data was discussed at the 32nd North American Cystic Fibrosis Conference, in a poster presentation titled “…

Vertex Pharmaceuticals’ VX-659 or VX-445 in combination with Kalydeco (ivacaftor) and tezacaftor (VX-661) can improve the lung function of patients with cystic fibrosis (CF) with one or two copies of the CFTR F508del mutation, Phase 2 data shows. Updated results of the studies were discussed…

Treatment with a combo of Proteostasis Therapeutics’ investigational CFTR modulator therapies — PTI-808, a  potentiator, plus PTI-801, a corrector — can promote significant improvements in lung function and reduction of sweat chloride in patients with cystic fibrosis (CF), Phase 1 data show. So far in the Phase…

AzurRx BioPharma is planning a multi-center Phase 2 trial to be conducted in the United States and Europe to evaluate its lead candidate MS1819-SD as a treatment for patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). This announcement follows the approval by the U.S. Food and…

Mutations in the CFTR gene that associate with severe disease can also lead to cardiac problems — specifically, the development of subclinical left ventricular dysfunction — in cystic fibrosis patients, a study suggests. The study, “Subclinical Left Ventricular Dysfunction is Influenced by Genotype Severity in Patients with Cystic…

Beginning Oct. 1, the cystic fibrosis (CF) targeted therapy Orkambi (lumacaftor/ivacaftor) will be included on Australia’s Pharmaceutical Benefits Scheme. The Australian government’s decision resulted from the completion of an agreement with Orkambi’s manufacturer, Vertex Pharmaceuticals, and was supported by the recent positive recommendation from the Pharmaceutical Benefits Advisory Committee…

Using a computer modeling method, a research team at the University of Copenhagen, Denmark, demonstrated some of the early developmental mechanisms involved in the formation of the pancreas’ network of ducts. The findings of the study, conducted in mice, offer new knowledge of how the pancreas works, and could…

Patients with cystic fibrosis (CF) have a greater risk of developing abnormalities in their retinal veins, which can impair visual acuity, a case report suggests. But researchers claim the higher risk is not due to one single factor, but to several contributing risk factors for vascular events. The study,…

Topline data from a Phase 2b trial showed that the investigative therapy acebilustat taken once daily can reduce the number and frequency of pulmonary exacerbations in patients with cystic fibrosis (CF). It did not, however, seem to affect patients’ overall lung function. Pulmonary exacerbations — temporary worsening of lung function — are…

Vertex Pharmaceuticals’ Symkevi (tezacaftor/ivacaftor) is one step closer to being approved in the European Union as a treatment for cystic fibrosis (CF) patients ages 12 or older who have certain CFTR genetic mutations. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency…