CAMBRIDGE (MASSACHUSETTS) (dpa-AFX) - Vertex Pharmaceuticals Inc. (VRTX) Thursday reported that its New Drug Application, or NDA, for its cystic fibrosis therapy Kalydeco has been accepted for priority review by the U.S. Food and Drug Administration, or FDA. The PDUFA target review date is April 18, 2012, the company added.
Further, the company announced the validation of its marketing authorization application, or MAA, by the European Medicines Agency, or EMA, with a regulatory review by the Committee for Medicinal Products for Human Use, or CHMP, now underway. The European application had earlier been accepted for accelerated assesment.
Cystic Fibrosis, which can result in chronic lung infections and progressive lung damage, often is caused by dysfunctional cystic fibrosis transmembrane conductance regulator, or CFTR, proteins. Kalydeco targets proteins which have a gating defect due to the G551D mutation. Approximately, 1200 people in the US, and 1000 in Europe, are believed to suffer from this condition.
In the U.S., Vertex Pharma has sought approval for Kalydeco as a target specifically for the G551D mutation, while in Europe approval is also sought for all other possible mutations. Chief Scientific Officer and Executive Vice President of Global Research and Development Peter Mueller commented 'If approved, KALYDECO will be the first treatment to target the underlying cause of CF. The commitments by the FDA and the EMA to expedite their reviews of our applications underscore the significant potential of KALYDECO to help people living with cystic fibrosis.'
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