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Vertex's Kalydeco Gets FDA Approval To Treat Cystic Fibrosis, Stock Up

Shares of Vertex Pharmaceuticals Inc. (VRTX) gained 8 percent Tuesday noon, after the US Food and Drug Administration approved Kalydeco as the first drug to treat the root cause of cystic fibrosis - a rare and incurable genetic disease that mostly affects the lungs, as also other bodily organs.

Vertex plans shipping Kalydeco, a 150-mg twice daily tablet, to pharmacy stores in the US this week.

Cystic fibrosis or CS, is a rare and malignant genetic disease caused by defective or missing cystic fibrosis transmembrane conductance regulator, or CFTR proteins due to mutations in the CFTR gene.

The FDA approval for Kalydeco means CF patients aged 6 and above, who have at least one copy of the G551D mutation in the CFTR gene, can be administered the drug. About 1,200 people in the US, or 4 percent of those with CF, are said to have this mutation.

Vertex discovered Kalydeco in a partnership with Cystic Fibrosis Foundation Therapeutics Inc., a nonprofit unit affiliated to the Cystic Fibrosis Foundation.

The FDA had accepted the Kalydeco application last December and had granted a six-month priority review. The approval was based on data from two Phase 3 studies of people with CF who have at least one copy of the G551D mutation.

Patients on Kalydeco showed strong and sustained improvements in lung function and other measures, compared to those on placebo. People on Kalydeco also experienced much fewer pulmonary exacerbations.

Adverse events for Kalydeco included headache, upper respiratory tract infection, stomach pain, and diarrhea. A Persist Phase 3 96-week extension study is underway to assess the long-term safety and durability of treatment with Kalydeco, Vertex noted.

Robert Beall, CEO of the Cystic Fibrosis Foundation, said, "We now have a medicine that treats the underlying cause of the disease in people with the G551D mutation. Kalydeco also provides us with a roadmap for exploring additional targeted approaches to treatment for all people with cystic fibrosis."

Vertex retains worldwide rights to develop and sell Kalydeco. An
European Medicines Agency accelerated review of Kalydeco is now in process after Vertex last October submitted a marketing authorization application.

The company is planning more studies this year to assess Kalydeco in children with CF aged 2 years and in people with CF who have the R117H mutation or gating mutations that were not probed prior Phase 3 studies.

Vertex is pinning hopes on becoming a viable drug company on the planned global launch of Kalydeco, and its hepatitis C drug Incivek that was approved in mid 2011.

VRTX is trading at $37.80, up $3.06 or 8.81%, on a volume of 5.7 million shares on the Nasdaq.

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