CF News

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CF News, Therapies
March 19, 2019

by Carey Goldberg, WBUR's "Common Health" [Note: This is a "good news" story. It describes the scientific road to the first drug that successfully attacks the underlying defect in cystic fibrosis, bringing dramatic improvements. But the drug appears to work for only 4% of cystic fibrosis...

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CF News, Therapies
March 19, 2019

Today the Cystic Fibrosis Foundation announced the expansion of its collaboration with Vertex Pharmaceuticals for the discovery and development of additional drugs aimed at treating the underlying cause of cystic fibrosis. The new program will support development of a potential new drug called VX-661, designed to...

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CF News, Therapies
March 19, 2019

In a widely expected move, the U.S. Food and Drug Administration has rejected a drug to treat cystic fibrosis made by Alnara Pharmaceuticals, which is owned by Indianapolis-based Eli Lilly and Co. The FDA has ordered additional clinical trials to further study the drug candidate,...

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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...