FDA Grants Priority Review for KALYDECO™ from Vertex Pharmaceuticals

Source: Vertex Pharmaceuticals press release

Vertex Pharmaceuticals Incorporated announced today that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for KALYDECO™ (ivacaftor) and granted the company’s request for six-month priority review.

KALYDECO targets the defective protein that causes cystic fibrosis (CF) in a subset of people with the disease. If approved, KALYDECO will be the first treatment to target the underlying cause of CF.

The FDA grants priority review to medicines that offer major advances in treatment or provide a treatment where no adequate therapy exists. A target review date of April 18, 2012, is set under the Prescription Drug User Fee Act (PDUFA) for the FDA’s approval decision, which is four months earlier than the standard review time of 10 months.

In addition, Vertex announced today that its marketing authorization application (MAA) for KALYDECO has been validated by the European Medicines Agency (EMA). Validation indicates that the application is complete and starts the regulatory review process by the Committee for Medicinal Products for Human Use (CHMP). Earlier this year, the EMA accepted Vertex’s request for accelerated assessment, which is granted to new medicines of major public health interest and shortens the EMA’s review time.

“If approved, KALYDECO will be the first treatment to target the underlying cause of CF,” said Peter Mueller, Ph.D., Chief Scientific Officer and Executive Vice President of Global Research and Development at Vertex. “The commitments by the FDA and the EMA to expedite their reviews of our applications underscore the significant potential of KALYDECO to help people living with cystic fibrosis.”

CF is caused by defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) proteins resulting from mutations in the CFTR gene. The absence of functional CFTR proteins results in poor flow of salt and water across cell membranes in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

In people with CF who have a gating defect, CFTR proteins are present at the cell surface but do not function properly. The most common gating defect is caused by the G551D mutation. Approximately 4 percent of those with CF, or about 1,200 people in the United States and 1,000 people in Europe, are believed to have this mutation. KALYDECO is designed to keep the CFTR channels at the cell surface open longer to improve the transport of chloride ions across the cell membrane in people who have gating mutations. The U.S. application seeks approval for KALYDECO in people with the G551D mutation. The application submitted in Europe includes a request for all gating mutations.

The regulatory submissions are supported by results from two Phase 3 studies, STRIVE and ENVISION, in which people with CF who had at least one copy of the G551D mutation and were treated with KALYDECO experienced rapid, significant and sustained improvements across a variety of disease measures, including lung function. The majority of adverse events associated with KALYDECO were mild to moderate in severity and non-serious. Fewer people in the KALYDECO treatment groups than in the placebo groups discontinued treatment due to adverse events. These data showed that treating the underlying cause of CF may improve outcomes for people with the disease.


KALYDECO (ivacaftor, VX-770) is Vertex’s lead medicine in development for the treatment of people with cystic fibrosis. Known as a CFTR potentiator, this oral medicine in development aims to help CFTR protein function more normally once it reaches the cell surface, which is believed to help hydrate and clear mucus from the airways. Vertex retains worldwide rights to develop and commercialize KALYDECO (kuh-LYE-deh-koh). The brand name KALYDECO has been approved by the EMA and provisionally approved by the FDA, but KALYDECO has not been granted marketing authorization or approval from any regulatory authority.