News

Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is safe for use in children with cystic fibrosis (CF) as young as age 6, data from a global Phase 3 clinical trial show. Based on these results, Vertex Pharmaceuticals, which markets Trikafta, will seek an expansion of the medication’s current U.S.

The U.S. Food and Drug Administration (FDA) has agreed to simplify Aridis Pharmaceuticals‘ planned Phase 2 trial of AR-501, an investigational inhaled treatment for chronic lung infections in patients with cystic fibrosis (CF). The decision was based on positive safety data from a Phase 1 trial in…

Savara Pharmaceuticals is stopping its Phase 2a exploratory clinical study of Molgradex (molgramostim nebulizer solution) to treat nontuberculous mycobacterial (NTM) lung infection in people with cystic fibrosis (CF), the company announced in a press release. The decision to discontinue the open-label ENCORE trial (NCT03597347) stems from an inability to achieve the study’s primary outcome. That goal…

A series of new research projects will seek to better understand how the immune system responds to transplanted organs, including the lungs. The goal is to find ways to increase the viability of organs after transplant, which could improve care for conditions like cystic fibrosis (CF). The projects are…

Pregnancy is generally safe for both women with cystic fibrosis (CF) and their child, but those with poorer lung function are at greater risk of having a premature or smaller baby, according to a U.K. population-based study. The study, “The outcome of pregnancy in women with cystic fibrosis:…

A new strategy for detecting very small concentrations of specific molecules could allow for better diagnosis and management of a variety of diseases, including cystic fibrosis (CF). The method was described in the journal Nature Communication by a team from the University of Leeds, U.K., in a study titled…

The U.S. Food and Drug Administration (FDA) is reviewing applications from Vertex Pharmaceuticals to expand the approval for three of the company’s cystic fibrosis (CF) therapies: Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor), and Kalydeco (ivacaftor). The three novel supplemental new drug applications…

ContraFect will receive funding from the Cystic Fibrosis Foundation to conduct preclinical research about the potential of direct lytic agents, an alternative to conventional antibiotics, to treat lung infections caused by Gram negative antibiotic-resistant bacteria, including Pseudomonas aeruginosa. While antibiotics have had a profound impact on…

People with cystic fibrosis (CF) in the U.K. have grown more numerous, older, and more diverse, according to the U.K. Cystic Fibrosis Registry’s annual data report for 2019. The report, meant to present a snapshot of both the gains as well as the challenges still faced by…

If Trikafta — Vertex Pharmaceuticals’ next-generation triple combination cystic fibrosis (CF) therapy — were to be available in Canada next year, the median age of CF patients could rise by 9.2 years and the number of deaths drop by 15% over the next 10 years, a forecast study reported.