News

Inhaled-IV Antibiotic Combo May Better Treat Acute Flares Than IV-only Mix

When given alongside the antibiotic Colistin (colistimethate), Cayston (aztreonam lysine), an inhaled antibiotic specifically targeting Pseudomonas aeruginosa, was superior to standard intravenous antibiotics at improving lung function and life quality among adults with cystic fibrosis (CF) and lung infections due to P. aeruginosa during an acute flare, a single-site…

Health Canada Giving Priority Review to Trikafta

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…

Arcturus to Develop ARCT-032 as RNA Therapy for CF

Arcturus Therapeutics has selected ARCT-032, an aerosolized RNA-based therapy, to be developed as a novel therapeutic candidate for cystic fibrosis (CF). The therapy will use the company’s LUNAR delivery technology that comprises a library of more than 150 proprietary fatty molecules specifically designed to deliver RNA-based therapies…

FDA Expands Labels for 3 Vertex CFTR Modulators

The U.S. Food and Drug Administration (FDA) has expanded the number of cystic fibrosis (CF)-related mutations considered responsive to and therefore eligible for Vertex Pharmaceuticals‘ three approved therapies. With these approvals, the labels will be expanded for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor), and…