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Vertex Announces Initiation Of Pivotal Phase 3 Program Of VX-809 In Combination With Ivacaftor For The Treatment Of People With Cystic Fibrosis Who Have Two Copies Of The F508del Mutation

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 in the U.S. 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.

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, Dr. Kauffman’s statements in the fourth paragraph of this press release and statements regarding (i) the clinical studies the company plans to conduct to evaluate VX-809 in combination with ivacaftor, (ii) the design of these studies, including the primary and secondary endpoints and the anticipated number of patients to be enrolled, (iii) the company’s expectations regarding when data will be available from these clinical trials, (iv) the potential submission of the NDA and MAA for the combination therapy and (v) the expectation that the data from the study in children 6 to 11 will be used for subsequent registration in the United States and the plan to continue discussions with European regulatory agencies for this age group. While the Company believes the forward-looking statements contained in this press release are accurate, those statements are subject to risks and uncertainties that could cause actual outcomes to vary materially from the outcomes referenced in the forward-looking statements. These risks and uncertainties include, among other things, the risks that efforts to develop VX-809 in combination with ivacaftor may not be successful because the results of the clinical trials described in this press release may not support registration or for technical, scientific or other reasons, that clinical trials may not proceed as planned due to drug supply, patient enrollment or other issues, and that an adverse event profile for VX-809 in combination with ivacaftor could be revealed in further nonclinical or clinical studies 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 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.

Conference Call Information

Vertex will host a conference call and webcast today, February 26, 2013 at 5:15 p.m. ET to review the initiation of the Phase 3 pivotal program studying the combination regimen of VX-809 and ivacaftor for F508del homozygous patients. The conference call will be webcast live, and a link to the webcast may be accessed from the ‘Vertex Events' page of Vertex's website at www.vrtx.com.

To listen to the live call on the telephone, dial 1-866-501-1537 (United States and Canada) or 1-720-545-0001 (International). To ensure a timely connection, it is recommended that users register at least 15 minutes prior to the scheduled webcast.

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