Author Archives: Marisa Wexler MS

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…

Health Canada is reviewing an application that seeks the approval of Trikafta, a triple combination reported to treat 90% of all with cystic fibrosis (CF), or those with least one F508del mutation. The request from Trikafta’s developer, Vertex Pharmaceuticals, covers eligible patients ages 12 and older, and has been granted…

A newly developed antibiotic, called T405, appears to effectively kill Mycobacteroides abscessus, a difficult-to-treat bacteria that can cause serious infections in people with lung conditions like cystic fibrosis (CF). The antibiotic was described in the study “Development of a penem antibiotic against Mycobacteroides abscessus,” published in Communications…

Children with cystic fibrosis (CF) who become infected with SARS-CoV-2 — the virus that causes COVID-19 — usually have a mild disease course, a new study reports. The findings also indicate that the risk of severe disease may be highest in children with CF who have poorer lung function…