Vertex receives first two Breakthrough Therapy Designations from US FDA

During its presentation at the JP Morgan annual health care conference, US drugmaker Vertex Pharmaceuticals (Nasdaq: VRTX) revealed that the US Food and Drug Administration had granted the first two Breakthrough Therapy Designations to it Kalydeco (ivacaftor) monotherapy and the combination regimen of VX-809 with ivacaftor for cystic fibrosis.

Enacted as part of the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA), Breakthrough Therapy Designation is intended to expedite the development and review of a potential new medicine if it is "intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development."

The FDA granted Breakthrough Therapy Designation to ivacaftor for potential additional indications beyond the currently approved use of this medicine for people with CF aged six years and older who have the G551D mutation. The designation for ivacaftor was based on data from clinical and pre-clinical studies, including Phase III 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 ivacaftor was based on the Phase II combination data announced in 2012. Multiple studies are currently underway in an effort to determine whether patients with other CFTR mutations may benefit from ivacaftor alone, and Vertex is also preparing to start a pivotal program of VX-809 in combination with ivacaftor in people who have two copies of the most common CF mutation (F508del).