VRTX 10-K & 10-Q changes, risk factors and insider trading
Vertex Pharmaceuticals Inc. / Ma · Nasdaq · Pharmaceutical Preparations · CIK 875320 · All filings on SEC.gov
At a glance
What changed in the latest 10-K
Risk Factors
New heading “Investing in our common stock involves a high degree of risk, and you should carefully consider the risks and”
New heading “uncertainties described below in addition to the other information included or incorporated by reference in this Annual”
New heading “Report on Form 10-K. If any of the following risks or uncertainties occur, our business, financial condition or results of”
New heading “operations would likely suffer, possibly materially. In that case, the trading price of our common stock could decline.”
New heading “Our business is substantially dependent on the success of our CF medicines.”
New heading “be materially harmed.”
New heading “We may not be able to increase or maintain CASGEVY product revenues.”
New heading “We are subject to pricing and reimbursement pressures that could have a material adverse effect on our business,”
New heading “revenues, and results of operations.”
New heading “Competing products and technological advances from our competitors may negatively affect our business and market”
New heading “If we discover safety or efficacy issues with any of our products, commercialization efforts for the product could be”
New heading “negatively affected, the approved product could lose its approval, and our business could be materially harmed.”
New heading “The data from our product development activities may not support advancement or regulatory approval of our product”
New heading “candidates, or label expansions for our marketed products, or provide sufficient data to support the successful”
New heading “commercialization of our approved products.”
New heading “If we fail to successfully conduct our clinical activities, our clinical trials or future regulatory approvals may be delayed or”
New heading “The extensive regulatory framework governing the health care industry could adversely affect our ability to obtain”
New heading “approval and market our medicines and failure to comply with these regulations could result in fines, penalties or other”
New heading “non-monetary remedies.”
New heading “Government Regulation.””
New heading “Commercialization of our products requires that we operate in compliance with applicable health care laws, including”
New heading “laws regulating promotional activities, prohibiting fraud and abuse and requiring reporting of government pricing”
New heading “If we are unable to obtain, maintain and enforce our intellectual property rights, our business could be harmed.”
New heading “If we are not able to operate without infringing upon intellectual property rights of third parties, our business could be”
New heading “We are subject to various and evolving laws and regulations governing the privacy and security of personal data.”
New heading “including at our third-party providers.”
New heading “If we are unable to maintain and expand our supply chain and manufacturing capabilities, our ability to develop our”
New heading “product candidates and manufacture our products would be harmed.”
New heading “Reliance on third-party relationships could adversely affect our business.”
New heading “adversely affect our business.”
New heading “Our operations may be disrupted by the occurrence of a natural disaster, catastrophic event, or by other serious accidents”
New heading “occurring at our facilities.”
New heading “Strategic and Financial Risks”
New heading “Our business development strategy, including strategic transactions and collaborations, may not be successful, and there”
New heading “may be delays or failures in realizing the anticipated benefits of these activities.”
New heading “Our effective tax rate fluctuates, and changes in tax laws, regulations and treaties, unfavorable resolution to the tax”
New heading “positions we have taken, and exposure to additional income tax liabilities could have a material impact on our future”
New heading “taxable income.”
New heading “Changes in foreign currency rates, interest rate risks, the value of our investment portfolio, and inflation affect our results”
New heading “of operations and financial condition.”
New heading “Future indebtedness could materially and adversely affect our financial condition, and the terms of our credit agreements”
New heading “impose restrictions on our business.”
New heading “There can be no assurance that we will repurchase shares of common stock or that we will repurchase shares at favorable”
New heading “Our stock price is volatile.”
New heading “that may frustrate any attempt to remove or replace members of our board or to effectuate certain types of business”
New heading “combinations involving us.”
Removed heading “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.”
Removed heading “SUMMARY OF RISK FACTORS”
Removed heading “Risks Related to Pricing of Our Products”
Removed heading “Risks Related to Development and Clinical Testing of Our Products and Product Candidates”
Removed heading “Risks Related to Supply, Manufacturing and Reliance on Third Parties”
Removed heading “Risks Related to Financial Results and Holding Our Common Stock”
Removed heading “Risks Related to Our Business”
Removed heading “If we are unable to successfully develop and commercialize additional products, our business could be materially harmed.”
Removed heading “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.”
Removed heading “If we are unable to successfully develop, obtain approval and commercialize treatments for acute and neuropathic pain, our business could be materially harmed.”
Removed heading “If we are not successful in commercializing CASGEVY, our revenue growth could be limited and our business could be materially harmed.”
Removed heading “If our competitors bring products with superior product profiles to market, our products may not be competitive, and our revenues could decline.”
Removed heading “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.”
Removed heading “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.”
Removed heading “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.”
Removed heading “Risks Related to Pricing of Our Products”
Removed heading “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.”
Removed heading “We may experience pricing pressure on our products, which could reduce our revenues and future profitability.”
Removed heading “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.”
Removed heading “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.”
Removed heading “Insurance coverage and reimbursement of cell and genetic therapies is uncertain.”
Removed heading “Risks Related to Development and Clinical Testing of Our Products and Product Candidates”
Removed heading “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.”
Removed heading “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.”
Removed heading “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.”
Removed heading “Difficulty in enrolling patients could delay or prevent clinical trials of our product candidates, and ultimately delay or prevent regulatory approval.”
Removed heading “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.”
Removed heading “Risks Related to Government Regulation”
Removed heading “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.”
Removed heading “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.”
Removed heading “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.”
Removed heading “The regulatory approval process for our cell or genetic therapies involves additional consultations with regulatory agencies, costs, and potentially longer timelines as compared to those for small molecules.”
Removed heading “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.”
Removed heading “If we do not comply with laws regulating the protection of the environment and health and human safety, our business could be adversely affected.”
Removed heading “Risks Related to Supply, Manufacturing and Reliance on Third Parties”
Removed heading “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.”
Removed heading “Risks Related to Business Development Activities”
Removed heading “We face risks in connection with existing and future collaborations with respect to the development, manufacture and commercialization of our products and product candidates.”
Removed heading “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.”
Removed heading “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.”
Removed heading “Risks Related to Intellectual Property”
Removed heading “If our patents do not protect our products or 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.”
Removed heading “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.”
Removed heading “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.”
Removed heading “Risks Related To Our Operations”
Removed heading “We rely on third parties to carry out our operations. Failure to maintain our third-party relationships or challenges at or with these third parties could materially harm our business.”
Removed heading “If our facilities were to experience a catastrophic loss, our operations would be seriously harmed.”
Removed heading “Risks Related to Financial Results and Holding Our Common Stock”
Removed heading “Our stock price may fluctuate.”
Removed heading “Our effective tax rate fluctuates, and changes in tax laws, regulations and treaties, unfavorable resolution to the tax positions we have taken or exposure to additional income tax liabilities could have a material impact on our future taxable income.”
Removed heading “Our quarterly operating results are subject to significant fluctuation.”
Removed heading “We expect that results from our clinical development activities and the clinical development activities of our competitors will continue to be released periodically, and may result in significant volatility in the price of our common stock.”
Removed heading “Future indebtedness could materially and adversely affect our financial condition, and the terms of our credit agreements impose restrictions on our business, reducing our operational flexibility and creating default risks.”
Removed heading “Issuances of additional shares of our common stock could cause the price of our common stock to decline.”
Removed heading “There can be no assurance that we will repurchase shares of common stock or that we will repurchase shares at favorable prices.”
Largest changes
“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. …”see in full comparison
“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. …”see in full comparison
“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.”see in full comparison
“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.”see in full comparison
“approval and market our medicines and failure to comply with these regulations could result in fines, penalties or other”see in full comparison
“Cyber-attacks are increasing in their frequency, sophistication, and intensity, and are becoming increasingly difficult to detect. They are often carried out by well-resourced and skilled parties, including nation states, organized crime groups, “hacktivists” and employees or contractors acting carelessly or with malicious intent. …”see in full comparison
Full comparison: every changed paragraph (999)
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 occur, 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.
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.
SUMMARY OF RISK FACTORS
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:
Risks Related to Our Business and Products
•IfOur wesuccess aredepends unableon our ability to successfully develop and commercialize additional products, our business could be materially harmed.medicines.
We invest significant resources in research and development to discover and develop transformative medicines for
people with serious diseases. Product development is highly uncertain and expensive. Product candidates may appear
promising in research and development but may fail to reach commercial success for many reasons, including:
•the failure to obtain marketing approval;
•the inability to manufacture on economically feasible terms;
•the failure to gain and maintain market acceptance among physicians and patients or other members of the medical
community;
•the failure to obtain adequate pricing or reimbursement levels from third-party payors or foreign governments; and
•competition based on, among other factors, safety, efficacy, patient convenience, pricing and reimbursement.
•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.
•If we are unablenot able to successfully develop, obtain approvaldevelop and commercialize treatmentsadditional for acute and neuropathic pain,medicines, our business couldwould be materially harmed.
harmed.
Our business is substantially dependent on the success of our CF medicines.
Substantially all our net product revenues have been derived from the sale of our CF medicines. We may be unable to
sustain or increase revenues from sales of our CF medicines in the future for any number of reasons, including the potential
introduction of competitive products or the inability to successfully develop and commercialize next-generation medications
or medicines to treat people with CF who cannot benefit from our current CF medicines. Our concentrated source of revenue
increases the risks associated with potential manufacturing or supply disruptions, safety issues that may be identified with
respect to our CF medicines, and failure to gain and/or maintain market acceptance or adequate pricing or reimbursement for
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
expectations of investors, our business would be materially harmed and our ability to fund our operations could be adversely
affected.
•If we are notunable successfulto insuccessfully commercializing CASGEVY, our revenue growth could be limiteddevelop and commercialize medicines for acute and neuropathic pain, our business could be materially harmed.
be materially harmed.
A portion of the value attributed to our company by investors is based on the expected commercial success of
JOURNAVX for acute pain and on our development programs for both acute and peripheral neuropathic pain. JOURNAVX
may not gain or maintain market acceptance among physicians, patients, or payors due to various factors, including the
availability of lower-cost alternatives, and sales, marketing, pricing, and/or distribution challenges associated with
introducing a product into a highly competitive market. Furthermore, we may not succeed in developing JOURNAVX for
additional indications or in advancing other product candidates, including NaV1.8 or NaV1.7 inhibitors, for the treatment of
acute or peripheral neuropathic pain. Even if we obtain marketing approvals for these product candidates, they will face
significant competition and there can be no assurance of commercial success.
We may not be able to increase or maintain CASGEVY product revenues.
The future commercial success of CASGEVY depends on physicians, patients, or payors accepting it as medically
useful, cost-effective, ethical, safe, and preferred with respect to current and potential future competitive therapies, and on
payors providing adequate reimbursement. In addition to risks generally associated with the commercialization of medicines,
the cell collection processes, manufacturing and other procedures required to manufacture and administer CASGEVY are
more complex, resource-intensive, and operationally demanding than for small molecules. For example, the cost of
manufacturing CASGEVY as a percentage of revenue is significantly higher than for our CF medicines. Moreover, 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 those resulting from the myeloablative preconditioning regime. There can be no
assurance that we will be able to increase or maintain our revenues from CASGEVY in future periods.
•If our competitors bring products with superior product profiles to market, our products may not be competitive, and our revenues could decline.
•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.
•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.
•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.
Risks Related to Pricing of Our Products
•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.
•We may experience pricing pressure on our products, which could reduce our revenues and future profitability.
•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.
•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.
Management's Discussion & Analysis (MD&A)
New heading “Our discussion and analysis of our financial condition and results of operations for 2025 as compared to 2024 are”
New heading “discussed below. For a discussion of our financial condition and results of operations for 2024 as compared to 2023, please”
New heading “refer to Item 7, “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our 2024”
New heading “Annual Report on Form 10-K, except as set forth below.”
New heading “Select R&D Pipeline Programs”
New heading “Revenues by Geographic Location”
New heading “Intangible Asset Impairment Charge”
Removed heading “Our discussion and analysis of our financial condition and results of operations for 2024 as compared to 2023 are discussed below. For a discussion of our financial condition and results of operations for 2023 as compared to 2022, please refer to Item 7, “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our 2023 Annual Report on Form 10-K, except as set forth below.”
Removed heading “Contingent Consideration”
Removed heading “Interest Expense”
Removed heading “Contingent Consideration”
Largest changes
“We may borrow up to a total of $500.0 million pursuant to a revolving credit facility that we entered into in July 2022 and could repay and reborrow amounts under this revolving credit agreement without penalty. Subject to certain conditions, we could request that the borrowing capacity be increased by an additional $500.0 million, for a total of $1.0 billion. Negative covenants in our credit agreement could prohibit or limit our ability to access this source of liquidity. As of December 31, 2024, the facility was undrawn, and we were in compliance with these covenants.”see in full comparison
“Our business also requires ensuring appropriate manufacturing and supply of our products. As we advance our product candidates through clinical development toward commercialization and market and sell our approved products, we build and maintain our supply chain and quality assurance resources. We rely on a global network of third parties, including some in China, and our internal capabilities to manufacture and distribute our products for commercial sale and post-approval clinical trials and to manufacture and distribute our product candidates for clinical trials. …”see in full comparison
“As of December 31, 2024 and 2023, we had goodwill of $1.1 billion on our consolidated balance sheets. During 2024, we did not have any business development transactions accounted for as a business combination. Goodwill reflects the difference between the fair value of the consideration transferred and the fair value of the net assets acquired. Thus, the goodwill that we record is dependent on the significant judgments and estimates inherent in these fair values. We have one reporting unit for goodwill reporting purposes. We evaluate our goodwill for impairment on an annual basis, and more”see in full comparison
“•In January 2025, we entered into a collaboration agreement with Zai Lab Limited (“Zai”) for the development and commercialization of povetacicept in mainland China, Hong Kong SAR, Macau SAR, Taiwan region and Singapore. Under this collaboration, Zai will help advance the povetacicept clinical trials, make the regulatory submissions in these territories, and will be responsible for commercialization activities in these territories, if povetacicept becomes an approved product.”see in full comparison
“Our discussion and analysis of our financial condition and results of operations for 2024 as compared to 2023 are discussed below. For a discussion of our financial condition and results of operations for 2023 as compared to 2022, please refer to Item 7, “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our 2023 Annual Report on Form 10-K, except as set forth below.”see in full comparison
Full comparison: every changed paragraph (611)
OPERATIONS
Our discussion and analysis of our financial condition and results of operations for 2025 as compared to 2024 are
discussed below. For a discussion of our financial condition and results of operations for 2024 as compared to 2023, please
refer to Item 7, “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our 2024
Annual Report on Form 10-K, except as set forth below.
Our discussion and analysis of our financial condition and results of operations for 2024 as compared to 2023 are discussed below. For a discussion of our financial condition and results of operations for 2023 as compared to 2022, please refer to Item 7, “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our 2023 Annual Report on Form 10-K, except as set forth below.
We are a global biotechnology company that invests in scientific innovation to create transformative medicines for
people with serious diseases, with a focus on specialty markets. We have approved medicines for cystic fibrosis (“CF”),
sickle cell disease (“SCD”), transfusion dependent beta thalassemia (“TDT”), and acute pain, and we continue to serially
innovate and advance next-generation clinical and research programs in these areas. Our mid- and late-stage clinical pipeline
includes programs across a range of modalities in additional serious diseases, including IgA nephropathy, APOL1-mediated
kidney disease, neuropathic pain, type 1 diabetes, primary membranous nephropathy, autosomal dominant polycystic kidney
disease, and myotonic dystrophy type 1.
Collectively, our five CF medicines, led by TRIKAFTA/KAFTRIO, are being used to treat nearly three quarters of the
people with CF in the U.S., Europe, Australia, and Canada. ALYFTREK, our newest CF medicine, is approved in the United
States (the “U.S.”), the United Kingdom (the “U.K.”), the European Union (the “E.U.”), Canada, New Zealand, Switzerland,
Australia and Israel.
CASGEVY, our ex-vivo, non-viral CRISPR/Cas9 gene-edited cell therapy, is approved in the U.S., the E.U., the U.K.,
the Kingdom of Saudi Arabia (“Saudi Arabia”), the Kingdom of Bahrain (“Bahrain”), Qatar, the United Arab Emirates (the
“UAE”), Kuwait, Switzerland and Canada for the treatment of people 12 years of age and older with SCD or TDT.
JOURNAVX, our selective non-opioid NaV1.8 pain signal inhibitor, is approved in the U.S. for the treatment of people
with moderate-to-severe acute pain. We are continuing our commercial launch of JOURNAVX for eligible adults.
We are a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases, with a focus on specialty markets. We have seven approved medicines: five that treat the underlying cause of cystic fibrosis (“CF”), a life-threatening genetic disease, one that treats severe sickle cell disease (“SCD”) and transfusion dependent beta thalassemia (“TDT”), life shortening inherited blood disorders, and one that treats moderate-to-severe acute pain. Our clinical-stage pipeline includes programs in CF, SCD, beta thalassemia, acute and peripheral neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy and other autoimmune renal diseases and cytopenias, type 1 diabetes, myotonic dystrophy type 1, and autosomal dominant polycystic kidney disease.
In December 2024, the U.S. Food and Drug Administration (the “FDA”) approved ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor), our once-daily next-in-class triple combination for the treatment of people with CF 6 years of age and older, and our fifth CF medicine. Collectively, our five medicines, led by TRIKAFTA/KAFTRIO (elexacaftor/tezacaftor/ivacaftor and ivacaftor), are being used to treat nearly three quarters of the approximately 94,000 people with CF in the U.S., Europe, Australia, and Canada. Through approvals of new medicines, label expansions, and expanded reimbursement, we are focused on increasing the number of people with CF who are eligible and able to receive our medicines. In December 2024, the FDA approved the expanded use of TRIKAFTA for the treatment of people with CF 2 years of age and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (“CFTR”) gene or a mutation that is responsive to TRIKAFTA. With this approval, 94 additional non-F508del CFTR mutations have been added to the TRIKAFTA label, and approximately 300 additional people with CF in the U.S. are now eligible for TRIKAFTA. In addition, we are evaluating our CF medicines in additional patient populations, including younger children, with the goal of having small molecule treatments for all people who have at least one mutation in their CFTR gene that is responsive to our CFTR modulators. We also are pursuing messenger ribonucleic acid (“mRNA”) and genetic therapies for people with CF who do not make full-length CFTR protein and, as a result, cannot benefit from our current CF medicines.
CASGEVY (exagamglogene autotemcel), our ex-vivo, non-viral CRISPR/Cas9 gene-edited cell therapy, is approved in the U.S., the European Union (“E.U.”), the United Kingdom (“U.K.”), the Kingdom of Saudi Arabia (“Saudi Arabia”), the Kingdom of Bahrain (“Bahrain”), the United Arab Emirates (the “UAE”), Switzerland and Canada for the treatment of people 12 years of age and older with SCD or TDT. We estimate approximately 60,000 people with severe SCD or TDT are or could become eligible for CASGEVY in the U.S., Canada, Europe, Saudi Arabia, and Bahrain.
In January 2025, the FDA approved JOURNAVX, our selective non-opioid NaV1.8 pain signal inhibitor, for the treatment of people with moderate-to-severe acute pain. We have begun our commercial launch of JOURNAVX in the U.S. for eligible adults. In addition, we are enrolling and dosing patients in a Phase 3 clinical trial evaluating suzetrigine for the treatment of diabetic peripheral neuropathy, a common form of peripheral neuropathic pain. In December 2024, we announced Phase 2 clinical trial results showing that treatment with suzetrigine demonstrated a statistically significant and clinically meaningful within-group reduction in pain on the numeric pain rating scale for people with lumbosacral radiculopathy (“LSR”), a form of peripheral neuropathic pain. The clinical trial also included a placebo reference arm, which showed a similar within-group reduction. Suzetrigine was safe and generally well-tolerated in the Phase 2 clinical trial. We hypothesize that a high placebo response in this clinical trial led to a lack of separation of the suzetrigine and placebo response curves. We believe we can innovate in pain clinical trial design to better control the placebo effect, and succeed in pivotal development with suzetrigine. We plan to advance suzetrigine into pivotal development in LSR, pending discussions with regulators on trial design and the regulatory package.
$0.1
$0.1
December 31, 2025
December 31, 2024
Cystic Fibrosis
We expect that the number of people with CF taking our medicines will continue to grow through new approvals and
reimbursement agreements, treatment of younger patients, increased survival and expansion into additional geographies.
•ALYFTREK is reimbursed for eligible people with CF in the U.S., England, Ireland, Germany, Denmark, Northern
Ireland, Norway, Wales, Italy, Australia, New Zealand and Luxembourg. We are working to secure access for
eligible patients in additional countries.
We expect to grow our CF business by increasing the number of people with CF who are eligible and able to receive our medicines. We have revised estimates for the number of people with CF in the U.S., Europe, Australia, and Canada from approximately 92,000 to approximately 94,000 people. Additionally, we continue to secure formal reimbursement in multiple additional countries that collectively comprise approximately 15,000 additional people with CF. Approximately 10,000 of those additional people with CF are eligible for treatment with CFTR modulators. We previously served many of these markets through named patient sales.
Recent progress in activities expanding our CF business is included below:
•In December 2024, the FDA approved ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor), the once-daily next-in-class combination CFTR modulator for the treatment of people with CF 6 years of age and older who have at least one F508del mutation or another mutation in the CFTR gene that is responsive to ALYFTREK, which includes a total of 303 CFTR mutations. Regulatory submissions for ALYFTREK, including in the U.K., the E.U., Canada, Switzerland, Australia, and New Zealand, are currently under review.
•In December 2024, the FDA approved the expanded use of TRIKAFTA for the treatment of people with CF with 94 additional non-F508del CFTR mutations. TRIKAFTA is now approved in the U.S. for a total of 272 CFTR mutations. We have also submitted regulatory applications to the European Medicines Agency (“EMA”) for TRIKAFTA/KAFTRIO for the treatment of people with CF and rare responsive mutations.
•We entered into an extended long-term reimbursement agreement with NHS England providing access to KAFTRIO, SYMKEVI and ORKAMBI, and continued access to KALYDECO, for existing and future eligible CF patients in England. We have entered into similar reimbursement agreements in Wales, Northern Ireland and Scotland. These reimbursement agreements include access to any future license extensions of these medicines.
•KAFTRIO is reimbursed in all 27 countries of the E.U.
•In 2025, we recorded $115.8 million of CASGEVY product revenues. This reflects 64 patients receiving infusions
of CASGEVY in 2025, including 30 people infused in the fourth quarter. Globally, in 2025, 147 people with SCD or
TDT had their first cell collection for CASGEVY.
•As of the end of 2025, approximately 90 percent of people with SCD or TDT in the U.S. have reimbursed access to
CASGEVY, which is also reimbursed in the U.K., Italy, Austria, Denmark, Luxembourg, Saudi Arabia, the UAE,
Bahrain, and Kuwait. In January 2026, we secured reimbursed access to CASGEVY for eligible people with SCD in
•CASGEVY is now approved in the U.S., the E.U., the U.K., Saudi Arabia, Bahrain, the UAE, Canada and Switzerland for people 12 years of age and older with SCD or TDT.
•We have activated more than 50 authorized treatment centers globally, and more than 50 patients have initiated cell collection. We expect significant growth in the number of new patients initiating cell collection throughout 2025.
•We entered into a reimbursement agreement with NHS England for eligible people with SCD to access CASGEVY,Scotland, consistent with the reimbursement agreement reached in August 2024 with NHS England2025 for eligible people with TDT to access CASGEVY.TDT.
•We expect to begin global regulatory submissions for approvals for CASGEVY in children 5 to 11 years of age, in
the first half of 2026. The FDA awarded Vertex with a Commissioner’s National Priority Voucher for this pediatric
submission, indicating an accelerated timeline for review once the submission is complete.
•Since pharmacy availability in March 2025 through year-end 2025, more than 550,000 prescriptions for
JOURNAVX were written and filled across the hospital and retail settings in different acute pain conditions,
consistent with JOURNAVX’s broad label.
•We have secured access for JOURNAVX with all three national pharmacy benefit managers, and, as of January
2026, over 200 million individuals across commercial and government payers have coverage, representing two-
thirds of U.S. covered lives. In addition, 21 states provide coverage via Medicaid.
What changed in the latest 10-Q
Risk Factors
New heading “We may be unable to complete the Crinetics Acquisition, successfully integrate Crinetics’ business, or realize the”
New heading “potential commercial benefits of the strategic acquisition, which could adversely affect our business and financial”
Largest changes
“potential commercial benefits of the strategic acquisition, which could adversely affect our business and financial”see in full comparison
“We may be unable to complete the Crinetics Acquisition, successfully integrate Crinetics’ business, or realize the”see in full comparison
“the need to satisfy customary closing conditions, the need for antitrust and/or other regulatory approvals, as well as potential”see in full comparison
“disputes or litigation that may arise. We provide no assurance that the Crinetics Acquisition will occur or that the closing”see in full comparison
“savings or synergies, if any, from the acquisition, which could adversely affect our business and financial condition. Further,”see in full comparison
“conditions to the Crinetics Acquisition will be satisfied in a timely manner or at all. Our realization of the value from the”see in full comparison
Full comparison: every changed paragraph (30)
Report on Form 10-K for the year ended December 31, 2025, which was filed with the SEC on February 13, 2026. There
We may be unable to complete the Crinetics Acquisition, successfully integrate Crinetics’ business, or realize the
potential commercial benefits of the strategic acquisition, which could adversely affect our business and financial
condition.
Our inability to complete the Crinetics Acquisition or to successfully integrate the Crinetics business could have a
material adverse effect on our business. The Crinetics Acquisition may not be completed for a number of reasons, including
the need to satisfy customary closing conditions, the need for antitrust and/or other regulatory approvals, as well as potential
disputes or litigation that may arise. We provide no assurance that the Crinetics Acquisition will occur or that the closing
conditions to the Crinetics Acquisition will be satisfied in a timely manner or at all. Our realization of the value from the
Crinetics Acquisition relies on successful integration of its operations. We may not be able to integrate Crinetics’ business
successfully into our existing business, make Crinetics’ business profitable, retain key employees or realize anticipated cost
savings or synergies, if any, from the acquisition, which could adversely affect our business and financial condition. Further,
our ongoing business may be disrupted, and our management's attention may be diverted by integration activities. In addition,
the anticipated benefits of the Crinetics Acquisition depend on revenues from PALSONIFY and the commercial potential of
atumelnant. If PALSONIFY does not achieve the sales, market acceptance, or other commercial performance we expect, if
development of atumelnant is delayed or terminated, or if we fail to obtain approval or fail to successfully commercialize
atumelnant, we may not realize the expected revenue growth or income contribution from these assets on the anticipated
timeline, or at all, which could adversely affect our business and financial condition.
have been no material changes from the risk factors previously disclosed in the Annual Report on Form 10-K.
•our ability to continue to launch, commercialize and market our productsproducts, andincluding ourthe abilityanticipated tolaunch obtain label expansionsof
povetacicept for the treatment of IgAN, and our ability to obtain label expansions for existing therapies;
for existing therapies, including the anticipated launch of povetacicept for the treatment of IgAN;
regulatory approval, including our expectations regarding the FDA’s review of theour BLA for povetaciceptaccelerated onapproval anof
povetacicept;
expedited basis of six months from the date of FDA acceptance of the BLA;
•our expectations, plans and anticipated timeline for the pending Crinetics Acquisition, including regarding Crinetics’
business and operations, the commercial potential of PALSONIFY, and the anticipated potential of atumelnant and
Crinetics’ other pipeline assets;
•our plans to buildmaintain and maintainexpand our global supply chains and manufacturing infrastructure and capabilities, including
including for biologics, cell and gene therapies;
Management's Discussion & Analysis (MD&A)
New heading “Investment in External Innovation”
Largest changes
“finance the Crinetics Acquisition, and can be prepaid without penalty. We may also borrow up to a total of $500.0 million”see in full comparison
“the 2026 Term Loan, as defined below. The Crinetics Acquisition is not conditioned on our receipt of financing. We will”see in full comparison
“•The FDA cleared the Investigational New Drug Application for VX-017, our stem cell-derived, fully differentiated”see in full comparison
see in full comparisonNegativeandcovenantsthein2026our credit agreementRevolver could prohibit or limit our ability to accessthisthesesourcesources of liquidity.As of March
“31, 2026, the facility was undrawn, and we were in compliance with these covenants.”see in full comparison
Full comparison: every changed paragraph (205)
MarchJune 31,30, 2026
Recent and anticipated progress in activities expanding our CF business is included below:
•In the second quarter of 2026, we secured reimbursement for ALYFTREK in four additional countries, including
Spain, bringing the total number of countries where ALYFTREK is reimbursed to 25. We also signed a letter of
intent with the Pan-Canadian Pharmaceutical Alliance for reimbursement of ALYFTREK for eligible patients six
years of age and older in Canada.
•The U.S. Food and Drug Administration (the “FDA”) approved label extensions for ALYFTREK and TRIKAFTA,
expanding availability of these medicines to approximately 95% of all people with CF in the United States (the
“U.S.”). With these label extensions, approximately 800 people with CF in the U.S. are newly eligible for a
medicine that treats the underlying cause of CF.
•We secured reimbursement agreements for ALYFTREK in Scotland, Spain, Sweden, Switzerland, New Zealand,
Israel, and Finland, and we are working to secure access for eligible patients in additional countries.
•In the firstsecond quarter of 2026, we recorded $43$76.4 million of CASGEVY product revenues.revenues, representing a 78%
increase compared to the first quarter of 2026 and a 151% increase compared to the second quarter of 2025.
•The U.S. Food and Drug Administration (the “FDA”) approved CASGEVY in children two years of age and older
with SCD or TDT, making it the first genetic therapy indicated for children as young as two years of age for both
SCD and TDT. Approximately 5,500 patients with SCD or TDT may be eligible for treatment with CASGEVY for
the first time with this approval. We also completed regulatory submissions in the Kingdom of Saudi Arabia (“Saudi
Arabia”) and the United Kingdom for the treatment of children five to eleven years of age.
•WeIn May, we secured a pricing agreementreimbursement for CASGEVY for eligible patients 12 years and older with SCD or TDT in Germany, and we are
Germany. We are committed to working with government and reimbursement authorities globally to ensure
sustainable access for eligible patients.
working through final implementation to provide long-term reimbursed access to patients at a sustainable price.
•We completed the regulatory submission in the U.S. for approval of CASGEVY in children with SCD or TDT five
to less than twelve years of age. The FDA awarded a Commissioner’s National Priority Voucher for this pediatric
submission, indicating an accelerated timeline for review once the submission is accepted.
•Since the launch of JOURNAVX in March 2025, more than 1 million prescriptions have been filled for
JOURNAVX across the hospital and retail settings for a broad range of acute pain conditions. In the first quarter of
•In the second quarter of 2026, more than 350,000 prescriptions were filled, and we recorded $29$49.6 million of JOURNAVX product revenues.revenues, representing a 71%
increase compared to the first quarter of 2026 and a more than 300% increase compared to the second quarter of
2025.
•In the second quarter and first six months of 2026, approximately 535,000 and 900,000 prescriptions, respectively,
•We have reached an agreement with a major pharmacy benefit manager for Medicare Part D coverage for
JOURNAVX effective on May 1. This agreement adds approximately 10 million lives covered under Part D.
Twenty-two states provide coverage for JOURNAVX via Medicaid. In total, approximately 240 million individuals
have reimbursedbeen accessfilled tofor JOURNAVX across athe wide range of commercialhospital and governmentretail payers.settings.
•We have reached agreements with two additional major pharmacy benefit managers for Medicare Part D coverage of
JOURNAVX. As a result, seniors covered by three of the four major Medicare Part D pharmacy benefit managers
have reimbursed access. Twenty-three states provide coverage for JOURNAVX via Medicaid. In total,
approximately 260 million individuals have reimbursed access to JOURNAVX across a wide range of commercial
and government payers.
•Following positive results from the ALYFTREKPhase 3 clinical trial evaluating ALYFTREK in children with CF two to five years of age, we expect
to submit for global regulatory approvals in this age group in the first half of 2026. We continue to enroll and dose
patients in the pivotal clinical trial evaluating ALYFTREK in children with CF one to less than two years of age.
•Following positive results from the TRIKAFTA clinical trial in children one to less than two years of age, we have
begunyears submissionsof forage, we initiated global regulatory approvalssubmissions infor this age group.
Acute and Peripheral Neuropathic Pain
•During the second quarter of 2026, Health Canada accepted our new drug submission for suzetrigine for the
treatment of moderate-to-severe acute pain, and review is underway.
•The FDA accepted our biologics license application for accelerated approval of povetacicept for adults with IgAN
and assigned a PDUFA target action date of November 30, 2026. If approved, povetacicept will become the first
commercialized therapy in our emerging nephrology franchise.
•We have completed our regulatory submission for accelerated approval of povetacicept in adults with IgAN in Saudi
Arabia, and the Saudi Food and Drug Authority has granted Breakthrough Designation to povetacicept.
•Following positive results from the RAINIER Phase 3 clinical trial evaluating povetacicept in adults with IgAN, we
completed in March the submission of the rolling biologics license application (“BLA”) to the FDA for potential
accelerated approval in the U.S. We are using a Priority Review Voucher and therefore expect the FDA review of
this BLA to be expedited to six months from the date of the FDA’s acceptance of the BLA.
nephropathy (“pMN”), another B cell-mediated disease. We completed enrollmentthe inPhase 2B portion of the Phase 2 portion of the/3
Phase 2/3 OLYMPUS pivotal trial evaluating povetacicept in people with pMN, and we initiatedconfirmed the Phasedose 3selection portionfor the
VRTX insider buying and selling (Form 4)
Form 4 filings since 2026-04-11: 0 open-market purchases and 14 open-market sales (about $11.0M; 14 reported as made under a Rule 10b5-1 trading plan), across 24 filings with stock transactions. Awards, option exercises, tax withholding and gifts are listed but not counted.
| Trade date | Insider | Transaction | Shares | Price | Value |
|---|---|---|---|---|---|
| 2026-09-08 | Van Grunsven Jasper |
Grant/award | 8,682 | — | — |
| 2026-08-28 | Ambrose Kristen |
Grant/award | 5,000 | — | — |
| 2026-08-27 | Sachs Bruce I |
Gift | 6,000 | — | — |
| 2026-07-21 | Tatsis Ourania |
Grant/award | 8,251 | — | — |
| 2026-07-21 | Sachdev Amit |
Grant/award | 8,251 | — | — |
| 2026-07-02 | Tatsis Ourania |
Open-market sale |
1,500 | $524.99 | $787.5K |
| 2026-07-02 | Mckechnie Duncan |
Open-market sale |
1,541 | $519.00 | $799.8K |
| 2026-07-01 | Liu Joy |
Open-market sale |
828 | $502.71 | $416.2K |
| 2026-06-26 | Bozic Carmen |
Open-market sale |
596 | $482.50 | $287.6K |
| 2026-06-18 | Bozic Carmen |
Open-market sale |
1,020 | $462.17 | $471.4K |
| 2026-06-15 | Bozic Carmen |
Open-market sale |
4,062 | $450.00 | $1.8M |
| 2026-06-05 | Bozic Carmen |
Open-market sale |
1,745 | $450.00 | $785.2K |
| 2026-06-01 | Liu Joy |
Open-market sale |
828 | $439.91 | $364.2K |
| 2026-05-29 | Bozic Carmen |
Open-market sale |
1,974 | $450.00 | $888.3K |
| 2026-05-15 | Bunnage Mark E. |
Open-market sale |
33 | $453.45 | $15.0K |
| 2026-05-15 | Bozic Carmen |
Open-market sale |
1,354 | $453.45 | $614.0K |
| 2026-05-12 | Bozic Carmen |
Open-market sale |
6,988 | $450.00 | $3.1M |
| 2026-05-04 | Bhatia Sangeeta N. |
Open-market sale |
318 | $423.73 | $134.7K |
| 2026-05-01 | Upadhyay Suketu |
Disposition to issuer | 796 | — | — |
| 2026-05-01 | Thornberry Nancy |
Grant/award | 472 | — | — |
| 2026-05-01 | Thornberry Nancy |
Disposition to issuer | 398 | — | — |
| 2026-05-01 | Schneider Jennifer |
Disposition to issuer | 796 | — | — |
| 2026-05-01 | Schneider Jennifer |
Grant/award | 472 | — | — |
| 2026-05-01 | Mckenzie Diana |
Disposition to issuer | 796 | — | — |
| 2026-05-01 | Mckenzie Diana |
Grant/award | 943 | — | — |
| 2026-05-01 | Liu Joy |
Open-market sale |
1,104 | $425.02 | $469.2K |
| 2026-05-01 | Garber Alan M |
Grant/award | 472 | — | — |
| 2026-05-01 | Bhatia Sangeeta N. |
Grant/award |
943 | — | — |
Well-known investors holding VRTX (13F)
None of the 59 investors we track reported a position in their latest 13F.