Vertex Begins Phase 3 Study of Kalydeco and VX-809 — Targeting Most Common CF Mutation

February 27, 2013

Vertex Pharmaceuticals Inc. announced the initiation of international Phase 3 clinical trials of Kalydeco™ in combination with VX-809, a CF drug in development. The combined therapies are designed to treat the underlying cause of cystic fibrosis.  

The six-month studies will examine the combination treatment in people ages 12 and older who have two copies of the Delta F508 mutation of CF, the most common mutation in cystic fibrosis. About 50 percent of people with CF in the United States have two copies of the Delta F508 mutation.

The studies will be conducted at approximately 200 clinical trial sites in North America, Europe and Australia. In total, about 1,000 patients will be enrolled across the two studies, which will test two different doses.

The U.S. Food and Drug Administration (FDA) recently awarded a “Breakthrough Therapy Designation” to Kalydeco and VX-809. This new designation is intended to speed the development of select potential therapies that treat life-threatening diseases or conditions. As a result, the timeframe of the Phase 3 trial of Kalydeco and VX-809 has been considerably shortened in order to accelerate testing. Most Phase 3 trials are about one year long.

Both Kalydeco and VX-809 are designed to treat the root cause of CF — a faulty gene and its protein product, CFTR. In January 2012, the FDA approved Kalydeco when taken alone for people with the G551D mutation of CF ages 6 and older.

People with CF and their families who have questions about the Phase 3 trial of Kalydeco and VX-809 should speak with their CF health care providers or call Vertex Medical Information at 1-877-634-8789.

Read the press release from Vertex.