Author: BEF

by
CF News, Therapies
March 19, 2019

Vertex Pharmaceuticals Incorporated today announced positive results from a 24-week analysis of the ongoing Phase 3 ENVISION study of VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis (CF). ENVISION (n=52) was designed to evaluate VX-770 among children ages...

by
CF News, Therapies
March 19, 2019

The FDA’s Gastrointestinal Drugs Advisory Committee (GDAC) has voted to recommend non-approval of Eli Lilly’s Solpura (liprotamase), a non-porcine pancreatic enzyme replacement therapy, currently under Agency review for the treatment of exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis and pancreatectomy. During the meeting, the GDAC...

by
CF News, Therapies
March 19, 2019

PTC Therapeutics, Inc. announced today that it has completed enrollment of a Phase 3 clinical trial of ataluren, an investigational new drug, in patients with nonsense mutation cystic fibrosis (nmCF). The 48-week study is designed to determine whether ataluren can improve lung function in patients with...

by
CF News, Therapies
March 19, 2019

Pharmaceutical company Pharmaxis today announced significant results of pooled data from its two large scale six month Phase III trials of Bronchitol (inhaled mannitol) in people with cystic fibrosis. The combined results have been presented for the first time at the North American Cystic Fibrosis Conference...