News

Patients with cystic fibrosis (CF) who carry certain mutations in the HFE gene, which are also behind an iron overload disorder, have poorer lung function that declines faster and a higher risk of CF-related diabetes (CFRD) and intestinal blockage,…

The Anton Yelchin Foundation has donated $1 million to the University of Southern California (USC) Center for Cystic Fibrosis – Adult Care at Keck Hospital to thank the center for its efforts in managing the healthcare of the late American actor. Yelchin, who had cystic fibrosis (CF), was cared…

Positive data from preclinical and Phase 2a studies evaluating the investigational cystic fibrosis (CF) treatment RPL554 were presented at the 2018 North American Cystic Fibrosis Conference (NACFC) in Denver, Colorado (Oct. 18-20). RPL554, developed by Verona Pharma, is an inhaled potential therapy for CF and chronic obstructive pulmonary disease (COPD).

Galapagos and AbbVie agreed that AbbVie will now have full control of the cystic fibrosis (CF) drug discovery portfolio the two companies have developed together. This means that the exclusive global rights to the investigational therapeutic candidates for CF will be held by AbbVie alone. This decision comes after months…

Symkevi (tezacaftor/ivacaftor combo), by Vertex Pharmaceuticals, has been approved in the European Union for patients with cystic fibrosis (CF) ages 12 or older who carry F508del mutations. Importantly, the European Commission’s decision to grant marketing authorization makes Symkevi the first treatment in Europe for CF patients with only one F508del copy plus another mutation that confers minimal…

Vertex Pharmaceuticals announced it plans to request by year’s end that the label for Symdeko (tezacaftor/ivacaftor and ivacaftor), its recently approved disease-modifying therapy for cystic fibrosis (CF), be expanded to include children as young as 6 years old, the company announced in a recent investor report. It also plans to…

A Phase 1b/2a, dose-escalating clinical trial evaluating the effects of POL6014 in patients with cystic fibrosis (CF) was recently launched, according to Santhera Pharmaceuticals, developer of the investigational therapy.  The Phase 1b/2a…

Adding the investigational therapy VX-445 to tezacaftor (VX-661) and Kalydeco (ivacaftor) led to a roughly 10 percent improvement in the lung function of cystic fibrosis (CF) patients with F508del mutations in the CFTR gene in a proof-of-concept Phase 2 clinical trial. …

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate, or…

Once-daily acebilustat, an investigational oral therapy for cystic fibrosis (CF) under development by Celtaxsys, reduces the frequency and delays the onset of pulmonary exacerbations in CF patients, results from a Phase 2 study show. Patients with better lung function…