News

Vertex Pharmaceuticals is launching a clinical trial to test VX-522, its inhaled messenger RNA (mRNA) therapy for lung disease in cystic fibrosis (CF) patients who are not eligible for treatment with an existing CFTR modulator. The study is expected to open soon, and it will assess VX-522’s safety and…

People with cystic fibrosis (CF) who are treated with Orkambi and Symkevi (Symdeko) in real-world settings tend to experience less decline of lung function, though results may be more modest and variable than what was reported in clinical trials for these therapies, a new study reports. “The…

The first participant has been dosed in a Phase 1 clinical trial evaluating Sionna Therapeutics‘ SION-638, an investigational oral treatment for cystic fibrosis (CF). The trial aims to assess the safety and pharmacokinetics — the movement into, through, and out of the body — of SION-638 in healthy volunteers.

First Wave Biopharma has selected the initial sites for the Phase 2 clinical trial of a new adrulipase formulation to treat exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF). “We believe that our reformulation of adrulipase offers the potential to provide a dramatically improved treatment option…

The U.K. National Institute for Health and Care Research awarded £1.5 million (about $1.8 million) for research on an at-home monitoring system for cystic fibrosis (CF) patients. The study will start in early 2023 and enroll patients across the U.K., who will use small wearable devices to collect health…

CF Youth, a new exercise program created by Beam for children living with cystic fibrosis (CF), was launched on the company’s online platform. The initiative, created in collaboration with CF Yogi and Johns Hopkins Cystic Fibrosis Center, can be accessed by caregivers and healthcare providers. Regular exercise has been…

A 30-year-old woman with cystic fibrosis (CF) and poor lung function gave birth to a healthy child while being treated with Trikafta, reportedly the first such successful CF pregnancy in Switzerland. The mother’s percent predicted forced expiratory volume in one second (ppFEV1) — meaning her expected ability to…

First Wave BioPharma has asked the U.S. Food and Drug Administration (FDA) for permission to start a Phase 2 clinical trial that would test the company’s new formulation of adrulipase in people with cystic fibrosis (CF) who have exocrine pancreatic insufficiency. Submitting the application “is an important step…

The Phase 2a clinical trial testing Aridis Pharmaceuticals’ experimental inhaled therapy AR-501 for chronic lung infections in people with cystic fibrosis (CF) is fully enrolled. AR-501, designed to be given once a week, can be self-administered within minutes using a hand-held nebulizer that delivers the medication directly to…

The antibiotic Xenleta (lefamulin) was well tolerated in a Phase 1 clinical trial in adults with cystic fibrosis (CF) and showed a similar pharmacological profile to healthy volunteers. “We are excited to share positive topline results from this important study of Xenleta in patients with CF,” said Christine…