FDA’s First Breakthrough Designations Continue Vertex <em>Kalydeco</em>’s Regulatory Successes

FDA’s First Breakthrough Designations Continue Vertex Kalydeco’s Regulatory Successes

07 Jan 2013

• By Cathy Dombrowski

Label expansion efforts for Vertex’s cystic fibrosis therapy will be claims taken through FDA’s new review pathway; the initial product could reach more patients more quickly and serve as a guide to how the agency will handle drugs earmarked for streamlined development and approval.

Breakthrough therapy designations could allow Vertex Pharmaceuticals Inc. to rapidly expand Kalydeco labeling to cover as much as 60% of the 70,000 worldwide cystic fibrosis population.

The company revealed Jan. 6 it has received the first two designations under the breakthrough therapy approval process created by...