News

The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have accepted for priority review applications for a combination of tezacaftor and Kalydeco (ivacaftor) as treatment for a subset of cystic fibrosis (CF) patients, Vertex Pharmaceuticals announced. “If approved, the tezacaftor/ivacaftor combination treatment would become Vertex’s third medicine to treat the underlying cause of…

Some children with severe infections who require intravenous antibiotics can be treated safely at home, according to a review of previously published studies. The review in the journal The Lancet Infectious Diseases reported that none of the studies found that home-based treatment was riskier than hospital care. The review also…

Inhalation of hypertonic saline solution (HSS) by children with cystic fibrosis (CF) increased sputum production and improved pathogen identification. These results suggest that this simple procedure, if implemented routinely in doctors’ offices and clinics, would help patients who can’t secrete mucus spontaneously. The study, “Hypertonic Saline as a Useful Tool…

Fluidigm and Baylor Genetics have teamed up to develop a next-generation gene sequencing method that can quickly identify less common mutations of the gene associated with cystic fibrosis. Up to 2,000 mutations can cause CF. The new method will allow doctors to spot those that are less common, improving…

Magnetic resonance imaging (MRI) scans, already important to both doctors treating patients and researchers conducting trials, might soon distinguish diseased and healthy tissue in differing colors, improving efforts to map and potentially diagnose and monitor diseases such as cystic fibrosis. A method allowing for two contrasting agents to be visible on MRI scans, called Dual…

Scientists have long known that defects in the CFTR protein cause cystic fibrosis. Now they’ve discovered that the defective protein leads to faulty insulin signaling, which, in turn, can cause diabetes and also contribute to cystic fibrosis complications. Insulin is a hormone that regulates metabolism, or the conversion of food to…

Corbus Pharmaceuticals plans to start a Phase 2b clinical trial of anabasum as a cystic fibrosis treatment by the end of 2017. The decision follows positive results of a Phase 2 study, the company said. Anabasum was formerly known as JBT-101 or Resunab. A Phase 2a trial usually looks at a drug’s effectiveness…

An influential group of European cystic fibrosis experts has signed off on the design of a Phase 3 clinical trial of Sollpura (lipromatase) as a treatment for a CF-related digestive disorder known as exocrine pancreatic insufficiency. The European Cystic Fibrosis Society Clinical Trial Network’s approval will speed up the recruitment of…

Kate Farms, which specializes in plant-based, ready-to-use oral and tube feeding formulas, has rolled out its Core Essentials Peptide 1.5 product. The high-caloric, peptide-based formula contains no dairy, soy, gluten, or other common allergens. With its approval by the Healthcare Common Procedure Coding System (HCPCS), it is now…

Verona Pharma recently announced the company’s accomplishments in the last six months, including an update to the company’s clinical trial in cystic fibrosis (CF) and positive interim results for its investigational therapy RPL554. RPL554 is an inhaled dual inhibitor of two enzymes, phosphodiesterase 3 and 4, and was previously shown to…