News

Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor), a licensed medicine used to treat cystic fibrosis (CF) patients ages 6 and older, has now been approved by the U.S. Food and Drug Administration as the first medicine to treat CF in children ages 2-5. The approval is for children who have…

Mologic, an U.K.-based company developing personalized diagnostic devices, announced the beginning of a Phase 1/2 clinical trial to test its urine-based diagnostic tool, HeadsUp, to monitor pulmonary exacerbations in patients with cystic fibrosis (CF). The study is designed to identify biomarkers, present in urine of CF adult patients, that…

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…

A previously undiscovered and rare type of cell has been identified in the tissue lining the airway of the lungs and carrying high levels of the CFTR gene, the mutation of which is the underlying cause of cystic fibrosis (CF), a study reports. While the exact role of…

Cystic fibrosis is (CF) patients receiving higher doses of pancreatic enzyme replacement therapy (PERT) gain more weight, a recent report shows. PERT is a tablet that contains pancreatic enzymes. When taken with food, this therapy can improve the absorption of necessary nutrients. Many CF patients struggle with weight gain. This is…

The Q359K/T360K gene mutation, which is commonly found in Jewish patients of Georgian descent, is associated with a particularly severe cystic fibrosis (CF) phenotype, according to researchers. Their study, “The Q359K/T360K mutation causes cystic fibrosis in Georgian Jews,” was published in the Journal of Cystic Fibrosis.

A high number of sudden disease worsening episodes — pulmonary exacerbations — and associated increases in healthcare costs were observed in Medicaid patients with cystic fibrosis (CF), according to researchers. Their study, “The burden of cystic fibrosis in the Medicaid population,” was published in the journal ClinicoEconomics…

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…

Vertex Pharmaceuticals plans to conclude the enrollment of patients in its Phase 3 programs involving its triple combo therapies to treat cystic fibrosis (CF) in the second half of 2018, the company recently announced in a corporate update. This is earlier than expected for the four Phase 3 trials, which…

Babies with cystic fibrosis (CF) are more likely to be born preterm — less than 37 weeks of pregnancy — and have significantly lower birth weight, according to a large study from Denmark and Wales. The study “Impact of cystic fibrosis on birthweight: a population based study of children…