VERTEX PHARMACEUTICALS INC / MA
For the period ended Dec 31, 2025. Filed Feb 13, 2026.
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A company's yearly report to the SEC, with audited financial statements. It is the fullest account a company gives of its business, its finances and the risks it sees.
FY2025 · Filed Feb 13, 2026
What changed in Risk Factors?
Read the current filingPrevious report: 10-K
Period ended 2024-12-31; filed 2025-02-13.
0000875320-25-000053
Item 1A. Risk FactorsCurrent report: 10-K
Period ended 2025-12-31; filed 2026-02-13.
0000875320-26-000056
Item 1A. Risk FactorsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 383 previous paragraphs and 673 current paragraphs.
16 unchanged; 102 reworded; 408 added; 261 removed; 4 previous and 147 current paragraphs rearranged.
Tables are not compared: 0 previous and 0 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (408)
Current report
Our success depends on our ability to develop and commercialize additional medicines.
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We invest significant resources in research and development to discover and develop transformative medicines for
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•competition based on, among other factors, safety, efficacy, patient convenience, pricing and reimbursement.
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If we are not able to successfully develop and commercialize additional medicines, our business would be materially
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Our business is substantially dependent on the success of our CF medicines.
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sustain or increase revenues from sales of our CF medicines in the future for any number of reasons, including the potential
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introduction of competitive products or the inability to successfully develop and commercialize next-generation medications
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or medicines to treat people with CF who cannot benefit from our current CF medicines. Our concentrated source of revenue
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increases the risks associated with potential manufacturing or supply disruptions, safety issues that may be identified with
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respect to our CF medicines, and failure to gain and/or maintain market acceptance or adequate pricing or reimbursement for
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JOURNAVX for acute pain and on our development programs for both acute and peripheral neuropathic pain. JOURNAVX
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may not gain or maintain market acceptance among physicians, patients, or payors due to various factors, including the
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availability of lower-cost alternatives, and sales, marketing, pricing, and/or distribution challenges associated with
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introducing a product into a highly competitive market. Furthermore, we may not succeed in developing JOURNAVX for
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additional indications or in advancing other product candidates, including NaV1.8 or NaV1.7 inhibitors, for the treatment of
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acute or peripheral neuropathic pain. Even if we obtain marketing approvals for these product candidates, they will face
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significant competition and there can be no assurance of commercial success.
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We may not be able to increase or maintain CASGEVY product revenues.
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The future commercial success of CASGEVY depends on physicians, patients, or payors accepting it as medically
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useful, cost-effective, ethical, safe, and preferred with respect to current and potential future competitive therapies, and on
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payors providing adequate reimbursement. In addition to risks generally associated with the commercialization of medicines,
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the cell collection processes, manufacturing and other procedures required to manufacture and administer CASGEVY are
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more complex, resource-intensive, and operationally demanding than for small molecules. For example, the cost of
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manufacturing CASGEVY as a percentage of revenue is significantly higher than for our CF medicines. Moreover, market
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assurance that we will be able to increase or maintain our revenues from CASGEVY in future periods.
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We are subject to pricing and reimbursement pressures that could have a material adverse effect on our business,
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Revenues from our products depend, to a large degree, on the extent to which the products are purchased by customers,
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such as wholesalers, pharmacies, and hospitals, and reimbursed by third-party payors, such as government health programs,
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commercial insurers, and managed health care organizations. Increasingly, these third-party payors are becoming more
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critical in evaluating and reimbursing medicines. The containment of health care costs continues to be a priority for many
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governments, and drug pricing has been a focus in this effort. The U.S. federal government and state legislatures and foreign
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governments have shown significant and evolving interest in implementing cost-containment programs, including price
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controls, restrictions on reimbursement, value-based and reference pricing, compulsory licensing, including the pursuit of so-
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Removed paragraphs (261)
Previous report
Our business is subject to numerous risks and uncertainties, discussed in more detail in the following section. These risks include, among others, the following key risks:
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•If we are unable to successfully develop and commercialize additional products, our business could be materially harmed.
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•If we are unable to sustain and grow revenues from sales of our CF medicines, our business would be materially harmed and the market price of our common stock would likely decline.
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•If we are not successful in commercializing CASGEVY, our revenue growth could be limited and our business could be materially harmed.
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•If our competitors bring products with superior product profiles to market, our products may not be competitive, and our revenues could decline.
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•If physicians and patients do not accept our products, or if patients do not remain on treatment or comply with their prescribed dosing regimen, our product revenues would decline in future periods.
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•Cell and genetic therapies face increased scrutiny from the public and medical communities and commercial success will depend, in part, upon the acceptance of those communities.
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•Government and other third-party payors seek to contain costs of health care through legislative and other means. If they fail to provide coverage and adequate reimbursement rates for our products, our revenues will be harmed.
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•We may experience pricing pressure on our products, which could reduce our revenues and future profitability.
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•Current health care laws and regulations in the U.S. and future legislative or regulatory reforms to the U.S. health care system may affect our ability to commercialize our marketed products profitably.
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•We have experienced challenges commercializing products outside of the U.S., and our future revenues will be dependent on our ability to obtain adequate reimbursement for our products in ex-U.S. markets.
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•Insurance coverage and reimbursement of our cell or genetic therapies is uncertain.
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Risks Related to Development and Clinical Testing of Our Products and Product Candidates
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•Our product candidates remain subject to clinical testing and regulatory approval, and our future success is dependent on our ability to successfully develop additional product candidates for both CF and non-CF indications.
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•If we are unable to obtain or are delayed in obtaining regulatory approval, we may incur additional costs, experience delays, or be unable to commercialize our product candidates.
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•If clinical trials are prolonged or delayed, our development timelines for the affected development program could be extended, our costs to develop the product candidate could increase and the competitive position of the product candidate could be adversely affected.
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•Difficulty in enrolling patients could delay or prevent clinical trials of our product candidates, and ultimately delay or prevent regulatory approval.
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•Enrollment for clinical trials for our cell and gene therapies may face additional and unique challenges and adverse developments associated with these clinical trials could result in action by regulatory bodies, including revised requirements for approval.
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•If regulatory authorities interpret any of our conduct, including our marketing practices, as being in violation of applicable health care laws, including fraud and abuse laws, laws prohibiting false and misleading promotion, disclosure laws or other similar laws, we may be subject to civil or criminal penalties.
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•If we fail to comply with our reporting and payment obligations under the Medicaid Drug Rebate Program or other governmental pricing programs in the U.S., we could be subject to additional reimbursement requirements, penalties, sanctions, and fines that could have a material adverse effect on our business, financial condition, results of operations and growth prospects.
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•If our processes and systems are not compliant with regulatory requirements, we could be subject to restrictions on marketing our products or could be delayed in submitting regulatory filings seeking approvals for our product candidates.
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•The regulatory approval process for our cell and genetic therapies involves additional consultations with regulatory agencies, costs, and potentially longer timelines as compared to those for small molecules.
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Risks Related to Supply, Manufacturing and Reliance on Third Parties
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•We rely on third parties to conduct pre-clinical work, clinical trials and other activities, and those third parties may not perform satisfactorily, including failing to meet established deadlines for the completion of such studies and/or trials or failing to satisfy regulatory requirements.
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Risks Related to Business Development Activities
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•We face risks in connection with existing and future collaborations with respect to the development, manufacture and commercialization of our products and product candidates.
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•Our ability to execute on our long-term strategy depends in part on our ability to engage in transactions and collaborations with other entities that add to our pipeline or provide us with new commercial opportunities.
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•We may not realize the anticipated benefits of existing or future acquisitions of businesses or technologies, and the integration following any such acquisition may disrupt our business and management.
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•If our patents do not protect our products and our products infringe third-party patents, we could be subject to litigation which could result in injunctions preventing us from selling our products, substantial damages, or circumvention of our patents by third parties.
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•Uncertainty over intellectual property in the pharmaceutical and biotechnology industry has been the source of litigation and other disputes that are inherently costly and unpredictable.
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•Failure to maintain our third-party relationships or challenges at or with these third parties could materially harm our business.
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Risks Related to Financial Results and Holding Our Common Stock
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•Our stock price may fluctuate and our quarterly operating results are subject to significant fluctuation.
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If we are unable to successfully develop and commercialize additional products, our business could be materially harmed.
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Reworded paragraphs (102)
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Investing in our common stock involves a high degree of risk, and you should carefully consider the risks and uncertainties described below in addition to the other information included or incorporated by reference in this Annual Report on Form 10-K. If any of the following risks or uncertainties actually occurs, our business, financial condition or results of operations would likely suffer, possibly materially. In that case, the trading price of our common stock could decline.
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Investing in our common stock involves a high degree of risk, and you should carefully consider the risks and
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We invest significant resources in the research and development of therapies for serious diseases and conditions, including CF, SCD, TDT, acute and peripheral neuropathic pain, IgAN, AMKD, T1D, DM1, and ADPKD. Product development is highly uncertain and expensive. Product candidates that may appear promising in research and development may fail to reach commercial success for many reasons, including:
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people with serious diseases. Product development is highly uncertain and expensive. Product candidates may appear
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•the failure to obtain marketing approval for the product candidate;
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•the inability to manufacture the product candidate on economically feasible terms;
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•the inability to manufacture on economically feasible terms;
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•the failure to gain and maintain market acceptance among physicians and patients or other members of the medical community; and
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•the failure to gain and maintain market acceptance among physicians and patients or other members of the medical
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•the failure to obtain market acceptance or adequate reimbursement levels from third-party payors or foreign governments for such product.
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•the failure to obtain adequate pricing or reimbursement levels from third-party payors or foreign governments; and
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Substantially all of our net product revenues have been derived from the sale of our CF medicines over the last several years. As a result, our business is dependent upon our ability to sustain and increase revenues from sales of our CF medicines. We seek to continue to increase our CF product revenue through serial innovation, including development and commercialization of next-generation CF medicines, extending access of CF medicines to younger children with CF, seeking additional approvals for our CF medicines in ex-U.S. markets and securing and maintaining adequate reimbursements for our CF medicines globally, and by developing a nebulized mRNA therapy for the more than 5,000 people with CF who do not make CFTR protein and cannot benefit from CFTR modulators.
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Substantially all our net product revenues have been derived from the sale of our CF medicines. We may be unable to
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If any of the above risks were to materialize, if we are otherwise unable to increase revenues from sales of our CF medicines, or if we do not meet the expectations of investors or public equity market analysts, our business would be materially harmed and our ability to fund our operations could be adversely affected.
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our CF medicines. If we are unable to sustain or increase revenues from sales of our CF medicines, or if we do not meet the
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•If we are unable to successfully develop, obtain approval and commercialize treatments for acute and neuropathic pain, our business could be materially harmed.
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If we are unable to successfully develop and commercialize medicines for acute and neuropathic pain, our business could
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We believe that a portion of the value attributed to our company by investors is based on our approved and potential treatments for acute and peripheral neuropathic pain. JOURNAVX, which was approved in January 2025 for moderate-to-severe acute pain in adults, may not gain or maintain market acceptance among physicians and patients or other members of the medical community. In addition to the risks normally associated with launching a new branded product, JOURNAVX will need to compete, and obtain reimbursement from third-party payors, in an acute pain market that largely consists of low-cost generic drugs, including opioids, non-steroidal anti-inflammatory drugs, acetaminophen and local anesthetics. Similarly, if we are successful in developing and obtaining approval for suzetrigine in peripheral neuropathic pain, this product will face competition from generic anticonvulsant and antidepressant drugs. If we are not able to successfully develop and commercialize treatments for acute and peripheral neuropathic pain, our future net product revenues and cash flows will be adversely affected and our business could be materially harmed.
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A portion of the value attributed to our company by investors is based on the expected commercial success of
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•global market acceptance continues to be dependent in part on the prevalence and severity of side effects associated with the procedure by which CASGEVY is administered, including the prevalence and severity of any side effects resulting from the myeloablative preconditioning regime.
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acceptance continues to be dependent in part on the prevalence and severity of side effects associated with the procedure by
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There also has been an increase in state legislation and regulations related to drug pricing and drug pricing transparency. In the U.S., various states, including Nevada, Maryland, Louisiana, New York, California, Washington, Massachusetts, New Jersey, Connecticut, Vermont, New Hampshire, Utah, Minnesota, Oregon, Colorado, New Mexico, Virginia, Maine, Texas, North Dakota, West Virginia, Florida, and New Jersey have passed legislation requiring companies to disclose extensive information relating to drug prices, drug price increases, and spending on research, development, and marketing, among other things. Although it is not always clear what states will do with the collected information, some laws were designed to obtain additional product discounts. Additionally, certain states have enacted laws establishing Prescription Drug Affordability Boards (“PDABs”). Some state PDABs either have the authority or have defined a pathway where they may be granted the authority to establish upper payment limits for prescription drugs, including Colorado, Maryland, Washington, and Minnesota. Under the Washington law, the PDAB cannot select for an affordability review drugs that are solely for the treatment of an orphan-designated disease or condition. In August 2023, the Colorado PDAB selected five drugs for an affordability review, including TRIKAFTA; later that year, it found TRIKAFTA to be not unaffordable, and thus not eligible for an upper payment limit. We cannot, however, predict whether future reviews by the Colorado PDAB, or any other PDAB, will come to the same conclusion about TRIKAFTA or any of our other therapies, or the amount of any potential upper payment limit. We may continue to see more state action requiring additional disclosures or other actions. In addition, we could see increased federal activity related to drug pricing and transparency requiring disclosures or other actions instead of, or in addition to, state requirements. Similar initiatives also are occurring in, or being considered by, some of our ex-U.S. markets, including Italy and Brazil.
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Colorado PDAB selected five drugs for an affordability review, including TRIKAFTA. Although the Colorado PDAB later
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In most ex-U.S. markets, the pricing and reimbursement of therapeutic and other pharmaceutical products is subject to governmental control and government authorities are making greater efforts to limit or regulate the price of drug products. The reimbursement process in ex-U.S. markets can take a significant time to conclude and reimbursement decisions are made on a country by country or region by region basis. Further, many ex-U.S. governments are introducing new legislation focusing on cost containment measures in the pharmaceutical industry. The final form of these laws and the relevant practical application is unknown at this time, but may lead to lower prices, paybacks or other forms of discounts or special taxes.
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markets vary widely and can take a significant time to complete, and reimbursement decisions are made on a country-by-
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Our CF medicines and CASGEVY treat life-threatening conditions and address relatively small patient populations, and our research and development programs are primarily focused on developing medicines to treat similar diseases. Both government and private payors are targeting these types of therapies, in some cases refusing to pay for them. We have experienced challenges in obtaining timely reimbursement for our products in various countries outside the U.S. Our future product revenues, including from TRIKAFTA/KAFTRIO, ALYFTREK, and CASGEVY, depend on, among other things, our ability to maintain reimbursement in ex-U.S. markets for our products. There is no assurance that coverage and reimbursement will be available outside of the U.S. for our approved or future therapies and, even if it is available, whether the timing or the level of reimbursement will be sufficient to allow us to market them. Adverse pricing limitations or a delay in obtaining coverage and reimbursement would decrease our future net product revenues and harm our business.
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environments. We have experienced challenges in obtaining timely reimbursement for our products in various countries
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Any new information regarding our products and product candidates, or competitive products or potentially competitive product candidates, can substantially affect investors’ perceptions regarding our future prospects. We, our collaborators, and our competitors periodically provide updates regarding drug and therapy development programs, typically through press releases, conference calls and presentations at medical conferences. These periodic updates often include interim or final results from clinical trials conducted by us or our competitors and/or information about our or our competitors’ expectations regarding regulatory filings and submissions as well as future clinical development of our products or product candidates, competitive products or potentially competitive product candidates. The timing of the release of information by us regarding our drug and therapy development programs is often beyond our control and is influenced by the timing of receipt of data from our clinical trials and by the general preference among pharmaceutical companies to disclose clinical data during medical conferences. In addition, the information disclosed about our clinical trials, or our competitors’ clinical trials, may be based on interim rather than final data that may involve interpretation difficulties and may in any event not accurately predict final results. The release of such information may result in volatility in the price of our common stock.
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our competitors regarding competitive products or potentially competitive product candidates can affect investors’
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•If we discover safety issues with any of our products or if we fail to comply with continuing U.S. and applicable foreign regulations, commercialization efforts for the product could be negatively affected, the approved product could lose its approval, and our business could be materially harmed.
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If we discover safety or efficacy issues with any of our products, commercialization efforts for the product could be
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Our products are subject to continuing regulatory oversight, including the review of additional safety information. Products are more widely used by patients once approval has been obtained and therefore side effects and other problems may be observed after approval that were not seen or anticipated, or were not as prevalent or severe, during pre-approval clinical trials or nonclinical studies. The subsequent discovery or appearance of previously unknown or underestimated problems with a product could negatively affect commercial sales of the product, result in restrictions on the product or lead to the withdrawal of the product from the market. Each of our CF products shares at least one active pharmaceutical ingredient with another of our products. As a result, if any of our CF products were to experience safety issues or labeling modifications, our other CF products may be adversely affected. For example, in December 2024, the FDA modified the labeling of TRIKAFTA by revising information regarding liver injury and liver failure and moving it from the “warnings and precautions” section to a “boxed warning” section, and included similar language in the ALYFTREK label. In SCD and TDT, as part of the FDA approval for CASGEVY, we are required to conduct two post-marketing requirement safety studies to assess the long-term risk of hematologic malignancies and off-target genome editing effects by CRISPR/Cas9. Negative or ambiguous results from these studies could have a significant impact on our ability to commercialize our products.
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unknown or underestimated safety or efficacy concerns with a product could negatively affect commercial sales of the
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The reporting of adverse safety events involving our products or public speculation about such events could cause our stock price to decline or experience periods of volatility. Our business also may be materially harmed by reduced coverage or reimbursement by payors, impaired sales of our products, denial or withdrawal of regulatory approvals, non-renewal of conditional regulatory approvals, required label changes or additional clinical trials, reputational harm, or government investigations or lawsuits brought against us.
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products, may also lead to recalls, denial or withdrawal of regulatory approvals, non-renewal of conditional regulatory
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Many companies in the pharmaceutical and biotechnology industries, including our company, have suffered significant setbacks in later-stage clinical trials even after achieving promising results in earlier-stage clinical trials. For example, the results from completed preclinical studies and clinical trials may not be replicated in later clinical trials, and ongoing clinical trials for our product candidates may not be predictive of the results we may obtain in later-stage clinical trials or of the likelihood of approval of a product candidate for commercial sale.
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trials may not be predictive of the results from later-stage clinical trials, or of the likelihood of approval of a product
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In addition, from time to time, we report interim, topline, and preliminary data from our clinical trials, which is based on a preliminary analysis of then-available data, and the results and related findings and conclusions are subject to change. Interim or preliminary data from a clinical trial may not be predictive of final results from the clinical trial and are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment and treatment continues and more patient data become available or as patients from our clinical trials continue other treatments for their disease. Topline data also remain subject to audit and verification procedures that may result in the final data being materially different from the preliminary data we previously published. As a result, topline data should be viewed with caution until the final data are available. If the interim, topline, or preliminary data that we report differ from actual results, or if others, including regulatory authorities, disagree with the conclusions reached, our ability to obtain approval for, and commercialize, our product candidates may be harmed, which could harm our business, operating results, prospects or financial condition.
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candidate for commercial sale. In addition, interim or preliminary data from a clinical trial may not be predictive of final
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Our business depends on relationships with third parties for a variety of functions, including activities critical to supply and manufacturing, commercialization, research and development, and technology. Failure by these parties to meet their contractual, regulatory, or other obligations, or any disruption in the relationship between Vertex and these third parties, could have an adverse effect on our business. Furthermore, these third parties are subject to their own unique operational and financial risks that are out of our control. When one of our third parties encounters financial, operational, or other difficulties, our business and results of operations could be negatively affected.
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other expenses incurred, which could have an adverse effect on our business, financial condition and results of operations,
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Results of our clinical trials and findings from our nonclinical studies, including toxicology findings in nonclinical studies conducted concurrently with clinical trials, could lead to abrupt changes in our development activities, including the possible cessation of development activities associated with a particular product candidate or program.
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In addition, results of our clinical trials and findings from nonclinical studies could lead to abrupt changes in our
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We have recently completed and/or have ongoing or planned clinical trials for several of our product candidates. The strength of our product portfolio and pipeline will depend in large part upon the outcomes of these clinical trials, including those evaluating TRIKAFTA/KAFTRIO and ALYFTREK in younger children with CF, VX-522 in CF, suzetrigine in peripheral neuropathic pain, VX-993 in acute and diabetic peripheral neuropathy, and zimislecel and VX-264 in T1D. Failure to advance product candidates through clinical development could impair our ability to ultimately commercialize products, which could materially harm our business and long-term prospects.
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not advance into further clinical studies. Failure to advance product candidates through clinical development would impair
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Any failure to obtain regulatory approvals for a product candidate would prevent us from commercializing that product candidate. Any delay in obtaining required regulatory approvals could materially adversely affect our ability to successfully commercialize a product candidate. Furthermore, any regulatory approval to market a product may be subject to limitations that we do not expect including with respect to the indicated uses for which we may market the product or required conditions of use. Any such limitations could reduce the size or demand of the market for the drug.
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could adversely affect our ability to successfully commercialize a product candidate.
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The regulatory approval process and clinical trial requirements for cell and genetic therapies can be more expensive and take longer than for other, better known or more extensively studied product candidates, and regulatory requirements governing cell and genetic therapy products have changed frequently and may continue to change in the future. For example, the FDA established the Office of Tissues and Advanced Therapies within its Center for Biologics Evaluation and Research (“CBER”) to consolidate the review of cell therapies and related products, and the Cellular, Tissue and Gene Therapies Advisory Committee to advise CBER on its review. These and other regulatory review agencies, committees and advisory groups, and the requirements and guidelines they promulgate, may lengthen the regulatory review process, require us to perform additional preclinical studies or clinical trials, increase our development costs, lead to changes in regulatory positions and interpretations, delay or prevent approval and commercialization of these treatment candidates or lead to significant post-approval limitations or restrictions.
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expensive and time-consuming than for other, better known or more extensively studied types of medicines, such as small
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The FDA and other regulatory agencies closely regulate the post-approval marketing and promotion of products to ensure that they are marketed only for the approved indications and in accordance or consistent with the provisions of the approved labeling. Although physicians are generally permitted, based on their medical judgment, to prescribe products for indications other than those approved by the applicable regulatory agency, manufacturers are prohibited from promoting such unapproved uses. We market our products to eligible people with CF, SCD, TDT, and acute pain for whom the applicable product has been approved and provide promotional materials and informational programs to physicians regarding the use of each product in these patient populations. These eligible people do not represent all people with CF, SCD, TDT, and acute pain. If a regulatory agency determines that our promotional materials, or other activities constitute promotion of unapproved uses or otherwise false and misleading promotion, it could request that we modify our promotional materials or other activities, conduct corrective advertising, or subject us to regulatory enforcement actions, such as the issuance of a warning or untitled letter, injunction, seizure, civil fines and criminal penalties. It also is possible that other federal, state, or foreign enforcement authorities might take action if they believe that the alleged improper promotion led to the submission and payment of claims for an unapproved use, which could result in significant fines or penalties under other statutory authorities, such as laws prohibiting false claims for reimbursement. Even if it is later determined we were not in violation of these laws, we may be faced with negative publicity, incur significant expenses defending our actions, and have to divert significant management resources from other matters.
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We market our products to health care providers and provide promotional materials and informational programs
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In the U.S., federal and state laws regulate financial interactions between pharmaceutical manufacturers and healthcare providers, require disclosure to government authorities and the public of such interactions, and mandate the adoption of compliance standards or programs. For example, the so-called federal “sunshine law” requires pharmaceutical manufacturers to report annually to CMS payments or other transfers of value made by that entity to physicians, physicians assistants, advanced practice registered nurses, and teaching hospitals. We also have similar reporting obligations with respect to financial interactions throughout the E.U. We expended significant efforts to establish, and are continuing to devote significant resources to maintain and enhance, systems and processes to comply with these regulations. Requirements to track and disclose financial interactions with health care providers and organizations increase government and public scrutiny of these financial interactions. As we commercialize products in areas with broader patient populations, we will have more interactions with a broader set of healthcare practitioners. Failure to comply with the reporting requirements could result in significant civil monetary penalties.
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We are required to track and disclose financial interactions with health care providers and health care organizations, which
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Due to evolving legal standards relating to the patentability, validity, and enforceability of patents covering pharmaceutical and biotechnological inventions and the scope of claims made under these patents, our ability to obtain, maintain and enforce patents is uncertain and involves complex legal and factual questions.
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complexity of the legal standards and factual questions relating to the patentability, validity, and enforceability of patents
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There has been, and we expect that there may continue to be, significant litigation and other disputes in the pharmaceutical industry regarding patents and other intellectual property rights. Litigation, arbitrations, administrative proceedings, and other legal actions with private parties and governmental authorities concerning patents and other intellectual property rights may be protracted, expensive, and distracting to management. Competitors may sue us as a way of delaying the introduction of our products or to remove our products from the market. Any litigation, including litigation related to Abbreviated New Drug Applications (“ANDA”), litigation related to 505(b)(2) applications, interference proceedings to determine priority of inventions, derivations proceedings, inter partes review, oppositions to patents in foreign countries, litigation against our collaborators or similar actions, may be costly and time consuming and could harm our business. We expect that litigation may be necessary in some instances to determine the validity and scope of certain of our proprietary rights. Litigation may be necessary in other instances to determine the validity, scope or non-infringement of certain patent rights claimed by third parties to be pertinent to the manufacture, use or sale of our products. Ultimately, the outcome of such litigation could adversely affect the validity and scope of our patent or other proprietary rights, hinder our ability to manufacture and market our products, or result in the assessment of significant monetary damages against us that may exceed amounts, if any, accrued in our consolidated financial statements.
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litigation related to Abbreviated New Drug Applications (“ANDA”), litigation related to 505(b)(2) applications, interference
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The laws of many foreign jurisdictions do not protect intellectual property rights to the same extent as in the U.S. We, like many companies in our segment of the pharmaceutical industry, have encountered challenges in protecting and defending such rights in foreign jurisdictions. Difficulties or preclusion from protecting our intellectual property rights in foreign jurisdictions, including through compulsory licensing, could substantially harm our business.
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Difficulties in, or preclusion from, protecting our intellectual property rights in foreign jurisdictions could substantially
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•We may be subject to claims by third parties asserting that our employees or we have misappropriated their intellectual property, or claiming ownership of what we regard as our own intellectual property.
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activities infringe their intellectual property rights or that our employees have misappropriated their intellectual property
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In addition to Broad, other third parties have filed patent applications claiming CRISPR/Cas9-related inventions and may allege that they invented one or more of the inventions claimed by the CVC Group. Thus, the USPTO may, in the future, declare an interference between certain CVC Group patent applications and one or more patent applications. Third parties could seek to assert their patents, if issued, against us based on our CRISPR/Cas9-based activities, including commercialization. Defense of these claims, regardless of their merit, could involve substantial litigation expense and could result in a substantial diversion of management and other employee resources from our business. In the event of a successful claim of infringement against us, we may have to pay substantial damages, obtain one or more licenses from third parties, pay royalties or redesign our infringing products, which may be impossible or require substantial time and monetary expenditure. In that event, we could be unable to further develop and commercialize CASGEVY or other products that we may develop using the CRISPR/Cas9 technology we license from CRISPR.
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redesign our infringing products, which may be impossible or require substantial time and monetary expenditure.
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•Decreased demand for our approved products, such as TRIKAFTA and ALYFTREK, or any product candidate for which we obtain marketing approval;
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products or any product candidate for which we obtain marketing approval, and may have a material adverse effect on our
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We have product liability insurance and clinical trial insurance in amounts that we believe are adequate to cover this risk. However, our insurance may not provide adequate coverage against all potential liabilities. If a claim is brought against us, we might be required to pay legal and other expenses to defend the claim, as well as pay uncovered damage awards and these damages could be significant and have a material adverse effect on our financial condition. Furthermore, whether or not we are ultimately successful in defending any such claims, we might be required to direct significant financial and managerial resources to such defense and adverse publicity is likely to result.
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insurance may not provide adequate coverage against all potential liabilities.
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We are subject to various and evolving laws and regulations governing the privacy and security of personal data, and our failure to comply could adversely affect our business, result in fines and/or criminal penalties, and damage our reputation.
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We are subject to various and evolving laws and regulations governing the privacy and security of personal data.
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We are subject to data privacy and security laws and regulations in various jurisdictions that apply to the collection, storage, use, sharing, and security of personal data, including health information, and impose significant compliance obligations. In addition, numerous other federal and state laws, including state security breach notification laws, state health information privacy laws and federal and state consumer protection laws, govern the collection, use, disclosure and security of personal information. The legislative and regulatory landscape for privacy and data protection continues to evolve, and there has been an increasing focus on privacy and data protection issues with the potential to affect our business. As we enter into new disease areas and jurisdictions both commercially and for clinical trials, we must continue to evaluate new and evolving privacy laws.
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jurisdictions related to the collection, storage, use, sharing, and security of personal data, including health information.
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For example, the E.U. General Data Protection Regulation (“GDPR”) went into effect in 2018 and has imposed new obligations on us with respect to our processing of personal data and the cross-border transfer of such data, including higher standards of obtaining consent, more robust transparency requirements, data breach notification requirements, requirements for contractual language with our data processors, and stronger individual data rights. Different E.U. member states have interpreted the GDPR differently and many have imposed additional requirements, which add to the complexity of processing personal data in the E.U. The GDPR also imposes strict rules on the transfer of personal data to countries outside the E.U., including the U.S. and the U.K., and permits data protection authorities to impose large penalties for violations of the GDPR. Similarly, other jurisdictions have either introduced or enacted legislation or executive orders restricting cross-border data transfers. These regulations restrict the transfer of certain types of data (e.g., sensitive personal data) or restrict the transfer of data to certain jurisdictions. The rules related to cross border data transfers continue to evolve based on court decisions and regulator guidance, which presents certain practical challenges to compliance. Regulators also continue to focus enforcement efforts on behavioral advertising and other online tracking technologies commonly used by companies. Compliance with these evolving rules is challenging, as country specific guidance and rules are continually changing and limited alternatives currently exist in the market. Compliance with the these laws and regulations is a rigorous and time-intensive process that may increase our cost of doing business or require us to change our business practices, and despite those efforts, there is a risk that we may be subject to fines and penalties, litigation, and reputational harm in connection with any activities falling within the scope of the GDPR or other privacy laws or regulations.
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personal information. Compliance with privacy laws and regulations is a rigorous and time-intensive process that may
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We must devote significant resources to understanding and complying with the changing landscape in this area. Failure to comply with data protection laws may expose us to risk of enforcement actions taken by data protection authorities, private rights of action in some jurisdictions, and potential significant penalties if we are found to be non-compliant. Failure to comply with the GDPR and applicable national data protection laws of European Economic Area member states could lead to fines of up to €20,000,000 or up to 4% of the total worldwide annual revenue of the preceding financial year, whichever is higher. Some of these laws and regulations also carry the possibility of criminal sanctions. For example, while we are not directly subject to the Health Insurance Portability and Accountability Act of 1996, as amended by the Health Information Technology for Economic and Clinical Health Act (“HIPAA”), we could be subject to penalties, including criminal penalties if we knowingly obtain or disclose individually identifiable health information from a HIPAA-covered health care provider or research institution that has not complied with HIPAA’s requirements for disclosing such information. In addition, the commercialization of cell and gene therapies requires the collection and processing of a greater amount of personal data than traditional therapies, potentially increasing risk. Furthermore, the number of government investigations and enforcement actions related to data security incidents and privacy violations, with a specific focus on online data sharing, continue to increase and government investigations typically require significant resources and generate negative publicity, which could harm our business and our reputation.
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incidents and privacy violations, particularly focused on online data sharing, continue to increase. Government investigations
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•We may face manufacturing, supply, and distribution difficulties, among other challenges, delays, or interruptions, including at our third-parties.
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We may face manufacturing, supply, and distribution delays, difficulties, and disruptions, among other challenges,
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We may face manufacturing, supply, and distribution difficulties, among other challenges, delays, or interruptions, including at our third-party providers.
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uncertainties described below in addition to the other information included or incorporated by reference in this Annual
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Report on Form 10-K. If any of the following risks or uncertainties occur, our business, financial condition or results of
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operations would likely suffer, possibly materially. In that case, the trading price of our common stock could decline.
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promising in research and development but may fail to reach commercial success for many reasons, including:
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expectations of investors, our business would be materially harmed and our ability to fund our operations could be adversely
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which CASGEVY is administered, including those resulting from the myeloablative preconditioning regime. There can be no
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found TRIKAFTA to be ineligible for an upper payment limit we cannot predict whether future reviews by the Colorado
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PDAB, or any other PDAB, will come to the same conclusion about TRIKAFTA or any of our other therapies, or the amount
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available, there is no assurance that the timing or level of reimbursement will be sufficient. Furthermore, many ex-U.S.
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governments are introducing new legislation focused on cost containment measures applicable to the pharmaceutical
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industry; such legislation, if finalized, could lead to lower prices, rebates or other forms of discounts or special taxes.
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negatively affected, the approved product could lose its approval, and our business could be materially harmed.
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management plan, and reductions in market acceptance. Each of our CF products shares at least one active pharmaceutical
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ingredient with another of our products. If any of our CF products were to experience safety issues or labeling modifications,
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our other CF products may be adversely affected. For example, in December 2024, the FDA required us to modify the
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TRIKAFTA label by revising information regarding liver injury and liver failure and moving that information from the
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“warnings and precautions” section to a “boxed warning” section; the FDA required similar language in the ALYFTREK
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The discovery of safety events involving our products or public speculation about such events could limit or reduce
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results from the clinical trial and are subject to the risk that one or more of the clinical outcomes may materially change as
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patient enrollment and treatment continues, more patient data become available, or as patients continue other treatments for
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development activities, including the possible cessation of development activities associated with a particular product
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our ability to commercialize products, which could materially harm our business, financial condition and long-term prospects.
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neuropathic pain label for suzetrigine at that time. The timing of the release of information by us regarding our product
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development programs is often beyond our control and is influenced by the timing of receipt of communications from
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molecules. Regulatory requirements governing cell and genetic therapy products have changed frequently and may continue
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regarding the use of each product in these patient populations. In jurisdictions where permitted, we also market our products
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to patients for whom the applicable product has been approved, as well as to their caregivers. If a regulatory authority
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uses or otherwise false and misleading, it could request that we modify or withdraw our promotional materials or issue
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our activities, seizing products, and imposing civil fines and criminal penalties. It is also possible that other federal, state, or
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foreign enforcement authorities might take action if they believe that the alleged conduct led to the submission and payment
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of claims for unapproved uses of our product, which could result in significant fines or penalties. Even if it is later determined
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we were not in violation of these laws, we may be faced with negative publicity, incur significant expenses defending our
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actions, and have to divert significant management resources from other matters.
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may increase government and public scrutiny of these financial interactions. Failure to comply with these reporting
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requirements could result in significant civil monetary penalties. As we commercialize products for new patient populations
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and in new geographies, we will have more interactions with a broader set of healthcare providers and we must continue to
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expend significant efforts to establish, maintain and enhance systems and processes to comply with laws and regulations
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covering pharmaceutical and biotechnological inventions and the scope of claims made under these patents, our ability to
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proceedings to determine priority of inventions, derivations proceedings, inter partes review, oppositions to patents in foreign
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countries, litigation against our collaborators, or similar actions, could harm our business.
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•prevalence and severity of adverse side effects;
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If we fail to scale our operations to accommodate growth, our business may suffer.
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A variety of risks associated with operating in foreign countries could materially adversely affect our business.
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If we fail to attract and retain skilled employees, our business could be materially harmed.