Alice Melão, MSc,  —

The approved CFTR modulators developed by Vertex Pharmaceuticals are too expensive for the clinical benefit they can provide to cystic fibrosis (CF) patients, according to the Institute for Clinical and Economic Review (ICER). ICER evaluated the effectiveness and costs of Symdeko (tezacaftor/ivacaftor), Orkambi (lumacaftor/ivacaftor), and…

Vertex Pharmaceuticals is launching Phase 3 clinical trials to evaluate the safety and effectiveness of its investigational therapy, VX-445, in combination with VX-661 (tezacaftor) and Kalydeco (ivacaftor) for cystic fibrosis (CF). The AURORA program will consist of two randomized, double-blind Phase 3 trials — AURORA F/MF…

Autogenic drainage physiotherapy can effectively improve the underlying lung mechanical impairment of cystic fibrosis (CF), a study shows. Respiratory physiotherapy is an important part of the treatment of patients with CF because it helps remove the mucus that accumulates in the lungs and that can ultimately lead to respiratory failure.

Quantification of two specific molecules in urine can help in the early detection of kidney damage triggered by treatment with aminoglycosides in patients with cystic fibrosis (CF), a study suggests. This finding was reported in the study “Urinary Biomarkers of Aminoglycoside-Induced Nephrotoxicity in Cystic Fibrosis: Kidney Injury…

The U.S. Food and Drug Administration has granted Fast Track Designation to Proteostasis Therapeutics’ triple combo treatment program for cystic fibrosis. The program includes the company’s amplifier, corrector, and potentiator of the CFTR gene — a combination designed to overcome the impact of the mutations that cause CF. The…

Organoid technology has created hopes of improved, personalized treatment for cystic fibrosis, but has also posed challenges and generated doubts. Organoids are three-dimensional, engineered cell structures built from stem cells that mimic the architecture and function of human tissue. These mini-organs have the potential to improve our knowledge of disease-related…

Key results of a Phase 1/1b clinical trial of Pulmatrix’s lead treatment for fungal infections in asthma and cystic fibrosis (CF) are expected by mid-2018, the company reports. The trial, which started in February, is focusing on pulmazole’s safety and patients’ ability to tolerate it. Pulmatrix expects the results to…

Anthera Pharmaceuticals is discontinuing the development of Sollpura (lipromatase) as a treatment for cystic fibrosis-related digestive problems. This follows the announcement of the latest data from the RESULT Phase 3 clinical trial (NCT03051490), which failed to demonstrate Sollpura’s effectiveness. The treatment was unable to significantly improve fat…

JHL Biotech plans to start a Phase 1 clinical trial this month of its cystic fibrosis (CF) therapy JHL1922, a biosimilar to Pulmozyme (dornase alfa) that is designed to improve lung function. The trial will be in the Netherlands, the company announced. The Dutch Healthcare Authority approved the trial…

Verona Pharma’s nebulized RPL554 can significantly improve lung function in patients with cystic fibrosis, according to top-line data from a Phase 2a clinical trial. The latest results revealed that single administration of both high and low doses of the investigative drug significantly increased patients’ average forced expiratory volume in…