Vertex Pharmaceuticals Incorporated

Item 1A. Risk Factors

The information presented below supplements the risk factors set forth in Part I, Item 1A. Risk Factors of our Annual Report on Form 10-K for the year ended December 31, 2024, which was filed with the SEC on February 13, 2025. There have been no material changes from the risk factors previously disclosed in the Annual Report on Form 10-K.

SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS

This Quarterly Report on Form 10-Q and, in particular, our Managements Discussion and Analysis of Financial Condition and Results of Operations set forth in Part I, Item 2, contain a number of forward-looking statements. Forward-looking statements are not purely historical and may be accompanied by words such as anticipates, may, forecasts, expects, intends, plans, potentially, believes, seeks, estimates, and other words and terms of similar meaning. Such statements may relate to:

our financial performance, including revenues, costs and expenses, taxes, and other gains and losses;

product development, including our development timelines, timing of data from our ongoing and planned clinical trials, regulatory authority filings and other submissions for our therapies, including potential to file for accelerated approvals, and communications with regulatory authorities;

our ability to continue to grow our CF business by increasing the number of people with CF eligible and able to receive our medicines through new approvals and reimbursement agreements, treatment of younger patients, increased survival, and expansion into additional geographies;

our ability to continue to launch, commercialize and market our products and our ability to obtain label expansions for existing therapies;

our ability to obtain and maintain adequate coverage, pricing, and reimbursement from third-party payors for our products;

the data that will be generated by ongoing and planned clinical trials, preclinical and nonclinical studies, and the ability to use that data to advance compounds, continue development or support regulatory filings;

our plans to continue investing in our research and development programs, including anticipated timelines for our programs, and our strategy to develop our pipeline programs, alone or with third party-collaborators;

our ability to use our research programs to identify and develop new product candidates to address serious diseases and significant unmet medical needs;

our beliefs regarding the approximate patient populations for the disease areas on which we focus;

plans for and prospects of our business development activities, including the potential benefits and therapeutic scope of our collaborations, our ability to integrate and continue operations of acquired businesses, and our ability to successfully capitalize on these opportunities;

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the establishment, development and maintenance of collaborative relationships, including potential milestone payments or other obligations, and other potential business development activities, including the identification of potential collaborative partners or acquisition targets;

our plans to build and maintain our global supply chains and manufacturing infrastructure and capabilities, including for biologics, cell and gene therapies;

our ability to expand and protect our intellectual property portfolio and otherwise maintain exclusive rights to products;

potentiaour expectations or beliefs regarding any legal proceedings in which we are involved, including any litigation, arbitration or other similar proceedings involving our products, product candidates or activities;

potential fluctuations in foreign currency exchange rates and the effectiveness of our foreign currency management program;

our expectations regarding cash generated by operations, our cash balance and expected generation and interest income;

our expectations regarding our provision for or benefit from income taxes and the utilization of our deferred tax assets; and

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our liquidity and our expectations regarding the possibility of raising additional capital.

Forward-looking statements are subject to certain risks, uncertainties, or other factors that are difficult to predict and could cause actual events or results to differ materially from those indicated in any such statements. These risks, uncertainties, and other factors include, but are not limited to, those described in our Risk Factors in Item 1A of our Annual Report on Form 10-K for the year ended December 31, 2024, which was filed with the SEC on February 13, 2025, and those described from time to time in our future reports filed with the Securities and Exchange Commission.

Any such forward-looking statements are made on the basis of our views and assumptions as of the date of the filing and are not estimates of future performance. Except as required by law, we undertake no obligation to publicly update any forward-looking statements. The reader is cautioned not to place undue reliance on any such statements.