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TheStreet Open House

Health Canada Approves KALYDECO™ (ivacaftor), The First Medicine To Treat The Underlying Cause Of Cystic Fibrosis In People With A Specific Genetic Mutation (G551D)

Vertex plans to initiate a pivotal program in early 2013 to evaluate a combination of VX-809, a CFTR corrector, and ivacaftor, a CFTR potentiator, in people with two copies of the F508del CFTR mutation, pending discussions with regulatory agencies. A Phase 2 study of VX-661, a second CFTR corrector, dosed in combination with ivacaftor for people with two copies of the F508del mutation is also ongoing, with final data expected in the first half of 2013.

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 non-profit 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 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 and other life-threatening diseases.

Founded more than 20 years ago in Cambridge, Mass., we now have ongoing worldwide research programs and sites in the U.S., U.K. and Canada. Today, Vertex has more than 2,000 employees around the world, and for three years in a row, Science magazine has named Vertex one of its Top Employers in the life sciences.

Vertex's press releases are available at www.vrtx.com.

About Vertex in Canada

In 2009, Vertex established a research and development site in Laval, Quebec through the acquisition of Virochem Pharma Inc. Vertex employs approximately 50 researchers and support staff in Laval and has established Commercial and Medical teams in Canada, including an expansion to support the launch of KALYDECO.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding (i) the Canadian approval and reimbursement process and Vertex’s plans to work closely with federal, provincial and territorial governments and private health insurers to bring KALYDECO to all eligible Canadians and (ii) Vertex's ongoing and planned clinical trials of ivacaftor alone and in combination with its CFTR corrector compounds, including its plans to initiate a pivotal program in early 2013 that is expected to evaluate VX-809 in combination with ivacaftor. While Vertex 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 the initiation of a pivotal program to evaluate VX-809 in combination with ivacaftor may be prevented or delayed and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company'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)

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