-Jeffrey Leiden, M.D., Ph.D., to become CEO in February 2012-
-Matthew Emmens elects to retire in May; will remain director for Vertex Board-
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that its Board of Directors has appointed Jeffrey Leiden, M.D., Ph.D., to the position of President and Chief Executive Officer (CEO), effective February 1, 2012. Dr. Leiden has been a Director on Vertex's Board since mid-2009 and brings to Vertex more than 20 years of scientific, commercial and financial experience in the pharmaceutical and biotechnology industries and clinical experience in academia as a practicing cardiologist and molecular biologist. Dr. Leiden began his employment with Vertex yesterday as part of the transition period before he formally takes the role of CEO on February 1. Until February 1, Matthew Emmens will retain the role of Chairman, President and CEO, at which time he will retire from these duties to serve as the company's Executive Chairman through May 2012. In May, Mr. Emmens plans to retire from his full-time employment with Vertex
but will continue to serve on the company's Board as a Director.
"Under Matt's leadership, Vertex established itself as a company capable not only of discovering important new medicines, but of successfully bringing those medicines to patients," said Dr. Leiden. "As a member of the Vertex Board, I have been extremely impressed with the company's ability to retain its clear focus on both groundbreaking science and improving the lives of people with serious diseases. It will be a privilege to lead Vertex at this exciting time and to further build the organization as we prepare for
the global launch of our second new therapy, advance our diverse pipeline and build value for shareholders in the years ahead."
Matthew Emmens, Chairman, President and CEO, added "I am proud of the
accomplishments we have made during my almost three years at Vertex and believe the company is well-positioned to bring forward additional innovative new medicines. Jeff brings significant and broad leadership experience to Vertex, and I am confident that his understanding of the company, combined with his unique blend of scientific, commercial and financial expertise, will help Vertex deliver on its goals in the future."
"The Board appreciates Matt's leadership to ensure the successful launch of INCIVEK and to prepare Vertex for continued growth," said Elaine Ullian, Chair of the Corporate Governance and Nominating Committee for Vertex's Board. "As we began planning for Matt's retirement, we carefully considered our vision for this new phase of the company and believe we found the ideal candidate in Jeff. His scientific background, together with his operational experience, will prove invaluable to Vertex at this important time."
Dr. Leiden, 56, has been a member of Vertex's Board since July 2009. Dr. Leiden is a Managing Director at Clarus Ventures, a life sciences venture capital firm he joined in 2006. In 2000, he joined Abbott Laboratories as President and Chief Operating Officer where he had responsibility for running Abbott's global pharmaceuticals business. While at Abbott, Dr. Leiden helped
launch multiple breakthrough medicines, including Humira for rheumatoid arthritis and other autoimmune diseases and Kaletra for HIV infection, among others. He also served as a member of the Board of Directors of Abbott Laboratories from 2001 to 2006.
Dr. Leiden began his career in academia as a practicing cardiologist and molecular biologist. From 1987 to 2000, Dr. Leiden held several academic appointments, including roles as Chief of Cardiology at the University of Chicago and Professor of Medicine at Harvard Medical School and Brigham and Women's Hospital. During his academic career, Dr. Leiden was also
involved in starting several biotechnology companies including Vical and Cardiogene.
Dr. Leiden held a number of board positions for pharmaceutical and biotechnology companies, including the role of nonexecutive
Vice Chairman for Shire plc. He was also a member of the Board of Directors of Millennium Pharmaceuticals, Inc.
Dr. Leiden received both his M.D. and Ph.D. degrees from the University of Chicago.

FDA Grants Priority Review for KALYDECO™ (ivacaftor), the First Potential Medicine to
Target the Underlying Cause of Cystic Fibrosis
-- Six-month review date of April 18, 2012 set by FDA --

-- European Medicines Agency accepts KALYDECO regulatory submission for accelerated assessment --
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S.
Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for KALYDECOTM (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.