cystic fibrosis

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…

In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to make clinical trials for new therapies more flexible and responsive to the needs of rare disease patients. From cystic fibrosis to epidermolysis bullosa, the FDA…

Ibuprofen does a good job of fighting bacteria in cystic fibrosis (CF) patients’ lungs, including two key threats, Pseudomonas aeruginosa and Burkholderia, a study shows. Researchers said the findings support ibuprofen’s use as an add-on therapy against bacterial infections in CF. The study, “Antimicrobial Activity of Ibuprofen against Cystic Fibrosis-Associated Gram-Negative…

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…

At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated 30 million Americans with rare diseases. Rep. Leonard Lance (R-New Jersey) and Sen. Amy Klobuchar (D-Minnesota) spoke to more…

Prepubescent children with cystic fibrosis (CF) have lower bone density compared to healthy children, and findings are comparable using two distinct bone density scanning methods, researchers report. The study that supports that conclusion, “Peripheral quantitative computed tomography detects differences at the radius in prepubertal children with cystic…

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…

Caregivers of people with rare diseases face emotional, financial, physical, and social strain and receive little outside support, a U.S. study found. The findings will be presented to Congress this spring along with policy recommendations to help support those caring for people with rare diseases. The report is titled “…

Pulmonary hypertension increases the risk of cystic fibrosis patients’ lung function worsening, but is not a predictor of reduced survival, a study reports. The research, ”Prognostic significance of pulmonary hypertension in patients with cystic fibrosis: A systematic review and meta-analysis,” was published in the journal Medicine.

Vertex Pharmaceuticals is launching a new Phase 3 trial to assess the triple combination of VX-659 plus tezacaftor (VX-661) and Kalydeco (ivacaftor) in cystic fibrosis (CF) patients with two copies of the F508del mutation in the CFTR gene. That mutation most common CF-causing genetic variant. The trial will enroll about 100…