Vertex's Orkambi Wins EU Nod To Treat Cystic Fibrosis In Kids
Zacks Investment Research | Jan 21, 2019 09:31PM ET
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) announced that the European Commission (EC) has granted an approval to a regulatory application seeking Orkambi’s label expansion for its use in children aged two to five years with cystic fibrosis (CF), who have two copies of the F508del mutation.
The EU approval was based on data from a phase III open-label safety study evaluating 60 patients, who showed good tolerance for a 24-week regimen under the influence of Orkambi and the safety profile was similar to that of patients aged six years or older.
Following this nod, Orkambi becomes the first and only approved medicine in Europe to treat CF in kids aged from two to five years. Approximately 1,500 children in Europe within the mentioned age group suffer this most common form of CF disease, thus making Orkambi eligible to treat an expanded patient population.
Orkambi is already approved in the United States and Europe for treating CF in patients aged six or above who have two copies of the F508del mutation. The drug was given a nod in the United States for the two to five-year age group last August.
Shares of Vertex were up 2.19% on Monday following this news. In fact, the stock has rallied 20.2% in the past year against the Original post
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