Vertex to file for EU approval of cystic fibrosis drug

8 November 2016

US drugmaker Vertex Pharmaceuticals (Nasdaq: VRTX) announced the results of a Phase III study of its cystic fibrosis drug, which it said will form the basis for a marketing application in Europe for a younger patient population, sending its shares up 5.4% to $83.22 by close of trading yesterday.

The trial of Orkambi (lumacaftor/ivacaftor) in children with cystic fibrosis (CF) ages six through 11 who have two copies of the F508del mutation met its primary endpoint of absolute change in lung clearance index (LCI2.5) through 24 weeks of treatment, demonstrating a statistically significant improvement in LCI2.5 among patients treated with Orkambi compared to placebo.

LCI is a sensitive measure of lung function in early CF disease and the European Medicines Agency (EMA) agreed to the primary endpoint for this study. In the first half of 2017, Vertex plans to submit a Marketing Authorization Application (MAA) line extension to the EMA for the use of Orkambi in this patient population.

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