BioCentury
ARTICLE | Company News

Vertex's CF products receive first breakthrough designations

January 8, 2013 2:10 AM UTC

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) said FDA granted Breakthrough Therapy Designation for its cystic fibrosis (CF) drug Kalydeco ivacaftor as monotherapy and for Kalydeco plus Vertex's VX-809. Vertex said the designations, which were created by the FDA Safety and Innovation Act, are the first to be issued by FDA. The designation commits FDA to collaborate with a sponsor to enable expedited development and review of compounds for serious or life-threatening diseases that show substantial improvements over existing treatments in early trials (see BioCentury, July 2, 2012).

Kalydeco is approved in the U.S. to treat CF in patients six years of age or older who have at least one copy of the G551D mutation in the CF transmembrane conductance regulator ( CFTR) gene. The small molecule potentiator of CF transmembrane conductance regulator (CFTR) is also in Phase III testing as a monotherapy in CF patients six years of age or older who have at least one copy of the R117H mutation, or who have at least one non-G551D CFTR gating mutation. The drug is also in Phase III testing in children ages two to five with CF who have a gating mutation. Data from the trials are expected in 2H13. This quarter, Vertex plans to start Phase III testing of Kalydeco plus VX-809, a small molecule CFTR corrector, in CF patients with two copies of the F508del mutation (see BioCentury, May 14, 2012). ...