News

The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended extending the licenses for Symkevi and Kalydeco, two cystic fibrosis (CF) treatments marketed by Vertex Pharmaceuticals, to allow these medications to be used in younger children. If approved by…

Moderna Therapeutics and Vertex Pharmaceuticals have entered a three-year research and licensing agreement aimed at developing gene-editing delivery techniques for the treatment of cystic fibrosis (CF). While Moderna will focus on ways to deliver gene-editing therapies to lung cells, Vertex will concentrate on other gene-editing components —…

The 7th edition of Bike 2 Breathe, an initiative aiming to raise funds and awareness for cystic fibrosis (CF), is planned to start on Sept. 21 in Boston. The event was created by Jerry Cahill, a CF patient and ambassador for the Boomer Esiason…

Adding pancreatic enzymes to “pre-digest” food was found to be an effective way to ensure nutrition in an infant with cystic fibrosis who was being fed through a feeding tube, according to a recent case report. The report, “Crushing pancreatic enzymes with enteral feeds in an extremely…

The European Medicines Agency (EMA) has validated an application that seeks to expand the approval of Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor) to treat cystic fibrosis (CF) in individuals 12 and older who have at least one copy of the F508del mutation…

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:…