Marisa Wexler, MS,  senior science writer—

Vertex Pharmaceuticals has made a $32 million purchase from Concert Pharmaceuticals related to future milestones from the 2017 agreement when Vertex purchased Concert’s investigational treatment for cystic fibrosis (CF), VX-561. According to Roger Tung, PhD, Concert’s president and CEO, the purchase will help Concert as it continues…

Combining AzurRx BioPharma‘s investigational therapy MS1819 with standard pancreatic enzyme replacement therapy (PERT) can improve fat absorption in cystic fibrosis (CF) patients with severe exocrine pancreatic insufficiency (EPI), interim data from a Phase 2 clinical trial indicate. “The overarching goal of our MS1819 program is to provide a safe and…

People with cystic fibrosis (CF) experience many barriers that limit their participation in physical activity, ranging from a lack of energy to low motivation to self-consciousness, a recent study highlights. A “tentative but important finding” from the study is that people with CF generally prefer at-home exercise over going…

The European Commission (EC) agreed to expand use of Vertex’s Kaftrio (ivacaftor/tezacaftor/elexacaftor), in combination with Kalydeco (ivacaftor), to include cystic fibrosis (CF) patients 12 and older who have at least one copy of the CFTR gene with an F508del mutation. The EC approved this combination therapy last year to treat people…

Blocking an enzyme called phosphodiesterase-4 (PDE4) may be useful in the treatment of dry mouth and cystic fibrosis (CF), new preclinical research suggests. The study, “Inhibition of cAMP-Phosphodiesterase 4 (PDE4) Induces Salivation in Mice,” is being presented virtually at the American Society for Pharmacology and Experimental…

A European Medicines Agency (EMA) committee favors extending the label for Kaftrio (ivacaftor/tezacaftor/elexacaftor), in combination with Kalydeco (ivacaftor), to treat cystic fibrosis (CF) patients 12 and older who have at least one copy of the CFTR gene with an F508del mutation. The European Commission (EC) approved the same combination last year…

Bronchitol (mannitol), an inhaled mucus-clearing treatment for cystic fibrosis (CF), is now commercially available in the U.S., according to Chiesi USA, which is marketing the therapy in the country. “We are excited to bring the first dry powder inhaled mucoactive agent to the U.S. for adults with CF,…

The first two patients have been dosed in the Phase 2b OPTION 2 extension study evaluating MS1819 in treating exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF), its developer, AzurRx BioPharma, announced in a press release. The thick mucus that accumulates in the organs of…

A Phase 1/2 clinical trial, called CYPHY, to evaluate the phage therapy YPT-01 in treating chronic Pseudomonas aeruginosa infections in people with cystic fibrosis (CF) has been initiated at Yale University. This double-blind, placebo-controlled study (NCT 04684641) aims to enroll 36 adult patients with these chronic infections. Additional…

Children with cystic fibrosis (CF) who live in more socioeconomically disadvantaged areas of the U.S. tend to have worse lung function and health outcomes than their counterparts in more affluent areas, a new study shows. The study, “The Association of Area Deprivation and State Child Health…