Therapies

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CF News, Therapies
March 19, 2019

Vertex Pharmaceuticals and the Cystic Fibrosis Foundation today announced positive results from an ongoing Phase 3 clinical trial of VX-770 for children age 6 to 11. VX-770 is an oral drug in development that targets the underlying cause of cystic fibrosis. Children who were on the drug...

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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...

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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...

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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...