Alice Melão, MSc,  —

Health Canada has approved the use of Kalydeco (ivacaftor) in toddlers age 1-2 years with certain genetic mutations that prevent the correct functioning of the CFTR protein, the therapy’s developer announced. With a pill taken twice a day, Kalydeco, by Vertex Pharmaceuticals, makes it possible to resolve the underlying…

New personalized strategies that can address the nutritional needs of cystic fibrosis patients may provide the best care and improve patient outcomes, a study suggests. The study, “A survey identifying nutritional needs in a contemporary adult cystic fibrosis cohort,” was published in the journal BMC Nutrition.

The Horizon 2020 research and innovation program of the European Union granted €6.8 million (approximately $7.7 million) to an international research team for the development of non-viral gene delivery technology. This new gene therapy strategy has the potential to improve the health of patients with genetic diseases worldwide, including…

The European Commission has approved the label extension of Orkambi (lumacaftor/ivacaftor) for the treatment of children ages 2-5 with cystic fibrosis (CF) due to F508del mutations, Vertex Pharmaceuticals, the therapy’s manufacturer, announced. Orkambi was previously approved in Europe to treat CF patients 6 and older with the…

Aridis Pharmaceuticals has enrolled the first healthy participant in its Phase 1/2a clinical trial to evaluate the antibacterial potential of its investigational candidate, AR-501 (gallium citrate), against chronic lung infections in patients with cystic fibrosis (CF). The study (NCT03669614) is expected to enroll approximately 48 healthy adult volunteers and…

Aridis Pharmaceuticals is planning to launch a Phase 1/2a clinical trial in the beginning of 2019 to evaluate the potential of its investigational antibacterial compound AR-501 (gallium citrate) in patients with cystic fibrosis (CF). This follows the approval of the Investigational New Drug application for AR-501 by…

GEn1E Lifesciences has added to its development pipeline an investigational non-antibiotic, antibacterial agent for treatment of Pseudomonas aeruginosa infections in people with cystic fibrosis (CF). The company established an exclusive option agreement with the University of Maryland, Baltimore (UMB) to license patent rights over the MUC1-ecto-domain (MUC1-ED) compound, currently…

The European Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Orkambi (lumacaftor/ivacaftor) as a treatment for children ages 2-5 with cystic fibrosis (CF) due to F508del mutations, Vertex Pharmaceuticals announced. CHMP’s positive opinion will be taken into consideration by the European Medicines Agency, which will…

A new test was able to assess various properties of the mucus that accumulates in the lungs of people with cystic fibrosis and other pulmonary diseases, and may help to find treatments — or a combination of treatments — for CF patients that can make coughing a more viable way…

Ten of 19 cystic fibrosis (CF) patients given antibiotics to manage pulmonary exacerbations did not achieve high enough blood concentrations of the antibiotic to sustain a therapeutic effect, results of single-site study show. This failure may be contributing to the development of antibiotic-resistant pulmonary infections, and to a worsening of…