Alice Melão, MSc,  —

Vertex Pharmaceuticals is launching a new Phase 3 trial to assess the triple combination of VX-659 plus tezacaftor (VX-661) and Kalydeco (ivacaftor) in cystic fibrosis (CF) patients with two copies of the F508del mutation in the CFTR gene. That mutation most common CF-causing genetic variant. The trial will enroll about 100…

Vertex Pharmaceuticals is offering the England arm of Britain’s National Health Service a package deal on the pricing of its current and future cystic fibrosis therapies. What the company describes as “a bold new portfolio approach” to pricing comes in the wake of the National Health Service balking at covering…

Santhera Pharmaceuticals has bought the rights to POL6014, a medication being developed by Polyphor to treat cystic fibrosis (CF) and other pulmonary diseases. Under terms of the global licensing agreement between the two Swiss companies, Santhera will develop, handle regulatory filings and market the investigative drug and derivate…

The sweat test, a clinical standard for diagnosing cystic fibrosis (CF), was found to be equally reliable for diagnosing CF in Down syndrome patients, according to a study published in the journal BMC Pulmonary Medicine. Previous case reports have suggested an association between CF and Down syndrome. Although rare, the…

An independent review board has given a green light to Anthera Pharmaceuticals continuing a Phase 3 clinical trial evaluating Sollpura’s (lipromatase’s) ability to treat a cystic fibrosis-related digestive disorder. It was the second time that the Data Monitoring Committee had decided that interim findings were good enough for the…

Changes in bacteria present in the lower respiratory tract during the first years of life may be indicative of early progression of lung disease in cystic fibrosis (CF) patients, according to researchers at the University of North Carolina School of Medicine. The finding was reported in the study, “…

ProQR Therapeutics and Galapagos are working together on a new approach to treating the tissue scarring seen in cystic fibrosis. The collaboration will take advantage of ProQR’s proprietary next-generation RNA technology to develop axiomer editing oligonucleotides that can fight the fibrosis-promoting agents Galapagos has identified. “In this collaboration we…

Some 3,400 European children whose cystic fibrosis (CF) is caused by the CFTR F508del mutation may now receive Orkambi (lumacaftor/ivacaftor), thanks to the European Commission’s decision to extend the therapy’s marketing authorization. The Jan. 10 extension, which applies to children aged 6 to 11, follows a positive recommendation issued…

A number of cystic fibrosis discoveries, therapy advances and other developments were reported by Cystic Fibrosis News Today during 2017. Now that the year is over, it’s time to review the articles that appealed most to our readers. Here are the 10 most-read articles of 2017, with a brief description of what…

Preventing bacteria from communicating with each other may be a way to bolster the effectiveness of current cystic fibrosis (CF) therapies, a study reports. According to researchers at the University of Montreal Hospital Research Centre (CRCHUM), the use of quorum-sensing inhibitors can help to prevent the harmful effects of…