Kalydeco

Treatment for Cystic Fibrosis

Update: Kalydeco (ivacaftor) Now FDA Approved - January 31, 2012

FDA Grants Priority Review for Kalydeco (ivacaftor), the First Potential Medicine to Target the Underlying Cause of Cystic Fibrosis

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dec 15, 2011 - 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.

About Kalydeco

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.

About Cystic Fibrosis

CF is a life-threatening genetic disease affecting approximately 30,000 people in the United States and 70,000 people worldwide. Today, the median predicted age of survival for a person with CF is approximately 38 years. According to the 2010 Cystic Fibrosis Foundation Patient Registry Annual Data Report, approximately 4 percent of the total CF patient population in the United States have at least one copy of the G551D mutation. The most common form of CF is caused by the F508del mutation, which is present in nearly 90 percent of people with the disease.

Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT)

Vertex initiated its CF research program in 1998 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. This collaboration was expanded to support the accelerated discovery and development of Vertex's CFTR modulators.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization. Many of the CF treatments available today benefited from Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit www.cff.org.

About Vertex

Vertex creates new possibilities in medicine. Our team discovers, develops and commercializes innovative therapies so people with serious diseases can lead better lives.

Vertex scientists and our collaborators are working on new medicines to cure or significantly advance the treatment of hepatitis C, cystic fibrosis, rheumatoid arthritis, epilepsy and other life-threatening diseases.

Founded more than 20 years ago in Cambridge, MA, we now have ongoing worldwide research programs and sites in the U.S., U.K. and Canada. Today, Vertex has more than 1,900 employees around the world, and Science magazine named Vertex number one on its 2011 list of Top Employers in the life sciences.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding (i) the FDA's target review date for the Kalydeco NDA, and (ii) the commitments by the FDA and EMA to expedite their reviews of our applications underscoring the significant potential of Kalydeco to help people living with cystic fibrosis. While the company believes the forward-looking statements contained in this press release are accurate, there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that Vertex could experience unforeseen delays in obtaining approval to market Kalydeco; that future scientific, clinical, competitive or other market factors may adversely affect the potential for therapy with Kalydeco and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through Vertex's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Contact: Vertex Pharmaceuticals Incorporated
Media:
Megan Goulart
Dawn Kalmar
617-444-6992
mediainfo@vrtx.com
or
Investors:
Michael Partridge, 617-444-6108
or
Lora Pike, 617-444-6755

Posted: December 2011

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