Now, FDA will work with Vertex to move these potentially lifesaving treatments through the FDA's approval process quickly and safely - and hopefully they'll be in the hands of patients in the near future.
The U.S. Food and Drug Administration has selected two Vertex Pharmaceuticals drugs to gain expedited review under a new program for “breakthrough” therapies.
The FDA will now speed its decision on whether or not to expand the uses of Vertex’s already-approved Kalydeco and its review of a combination therapy that pairs Kalydeco with Vertex’s experimental VX-809.
“Now, FDA will work with Vertex to move these potentially lifesaving treatments through the FDA's approval process quickly and safely - and hopefully they'll be in the hands of patients in the near future,” says Senator Michael Bennet, D-Colorado, who helped to get the breakthrough provision included in the FDA Safety and Innovation Act of 2012.
The breakthrough pathway is intended give the agency a new tool to expedite the development of therapies that show substantial promise in early clinical trials to treat serious or life-threatening diseases. But whether that intention will have a material impact on Vertex development activities, and subsequent regulatory submissions remains to be seen, the company says.
The designation requires that preliminary clinical evidence indicate that the drug may deliver a substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
Vertex says that the FDA granted the designation for Kalydeco based on potential additional indications beyond its currently approved use for people with cystic fibrosis ages 6 and older who have a genetic mutation known as G551D. The agency made the designation based on data from clinical and pre-clinical studies, including late-stage data in people with the G551D mutation and pre-clinical data in the G551D mutation as well as a number of other mutations.
The Breakthrough Therapy designation for the combination regimen of VX-809 with Kalydeco was based on mid-stage combination data announced in 2012, according to the company. Multiple studies are currently underway in an effort to determine whether patients with other CFTR mutations may benefit from Kalydeco alone, and Vertex is also preparing to start a pivotal program of VX-809 in combination with Kalydeco in people who have two copies of the most common CF mutation, F508del.
January 11, 2013