Vertex Reports First Quarter 2026 Financial Results
Key Terms
accelerated approval regulatory
cftr medical
crisper/cas9 medical
priority review voucher regulatory
fast track regulatory
orphan drug regulatory
priority medicines (prime) regulatory
– Total revenue of
– Povetacicept program continues rapid advancement: Completed rolling BLA submission for
– Continued progress across broad clinical-stage pipeline, including label expansion of eligible
“Vertex is off to a strong start in 2026, driven by leadership in cystic fibrosis; growth in sickle cell disease, beta thalassemia, and acute pain; as well as rapid pipeline progress,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “CASGEVY and JOURNAVX delivered more than 25 percent of our growth this quarter, underscoring the strength of the increasingly diversified revenue base. As we execute across the commercial portfolio and pipeline and build our fourth franchise in nephrology, Vertex is poised to continue to deliver for patients and create long-term value.”
First Quarter 2026 Results
Total revenue increased
Combined GAAP and non-GAAP R&D, Acquired IPR&D and SG&A expenses were
GAAP effective tax rate was
Non-GAAP effective tax rate was
GAAP net income was
Non-GAAP net income was
Cash, cash equivalents, and total marketable securities as of March 31, 2026, were
Full Year 2026 Financial Guidance
Vertex today reiterated full year 2026 financial guidance. Vertex’s total revenue guidance of
Vertex’s financial guidance is summarized below:
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Current FY 2026 |
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Previous FY 2026 |
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Total revenue |
Unchanged |
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Non-CF product revenue |
Unchanged |
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Combined GAAP R&D, AIPR&D and SG&A expenses * |
Unchanged |
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Combined non-GAAP R&D, AIPR&D and SG&A expenses* |
Unchanged |
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Non-GAAP effective tax rate |
Unchanged |
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*The difference between the combined GAAP R&D, AIPR&D and SG&A expenses and the combined non-GAAP R&D, AIPR&D and SG&A expenses guidance relates primarily to |
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**Combined GAAP and non-GAAP R&D, AIPR&D and SG&A expenses guidance includes approximately |
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Key Business Highlights
Marketed Products
Cystic Fibrosis (CF) Portfolio
Vertex has worked for more than 20 years to discover and develop medicines to treat the underlying cause of CF. Vertex CFTR modulators can treat approximately 95 percent of all people living with CF in core markets, including patients as young as one month old. ALYFTREK, the newest marketed CFTR modulator, is approved in the
-
The
U.S. Food and Drug Administration (FDA) recently approved label extensions for ALYFTREK and TRIKAFTA, expanding availability of these medicines to approximately95% of all people with CF inthe United States . This label expansion was supported by clinical and/or in vitro data from 564 variants demonstrating response to ALYFTREK and 521 variants demonstrating response to TRIKAFTA. With this approval, approximately 800 more people with CF in theU.S. are now eligible for the first time for a medicine that treats the underlying cause of their disease. -
Vertex recently secured reimbursement agreements for ALYFTREK in
Scotland ,Spain ,Sweden ,Switzerland ,New Zealand ,Israel , andFinland and is working to secure access for eligible patients in additional countries. - Following recently reported positive results from the study of ALYFTREK in children ages two to five years, Vertex is on track to submit for global regulatory approvals in the first half of 2026. Vertex continues to enroll and dose the pivotal study of ALYFTREK in children ages one to less than two years.
- Following recently reported positive results from the study of TRIKAFTA in children ages one to less than two years, Vertex has begun submissions for global regulatory approvals for TRIKAFTA in this age group.
CASGEVY for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)
CASGEVY is a non-viral, ex vivo, CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT that has been shown to reduce or eliminate vaso-occlusive crises (VOCs) for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved in the
-
Vertex recorded first quarter 2026 CASGEVY revenue of
.$43 million -
Vertex recently secured a pricing agreement for CASGEVY for eligible patients with SCD or TDT in
Germany . Vertex is now working through final implementation to provide long-term reimbursed access to patients at a sustainable price. -
Vertex completed the regulatory submission in the
U.S. for approval of CASGEVY in children ages 5 to less than 12 years old with SCD or TDT. The FDA awarded Vertex a Commissioner’s National Priority Voucher for this pediatric submission, indicating an accelerated timeline for review once the submission is accepted.
JOURNAVX (suzetrigine) for the treatment of moderate-to-severe acute pain
JOURNAVX is a first-in-class, oral, selective, non-opioid NaV1.8 pain signal inhibitor, approved in the
-
Since the launch of JOURNAVX in March 2025, more than 1 million prescriptions have now been filled for JOURNAVX across the hospital and retail settings for a broad range of acute pain conditions. In the first quarter of 2026, more than 350,000 prescriptions were filled, and Vertex recorded revenue of
.$29 million - The Centers for Medicare and Medicaid Services (CMS) have approved the inclusion of JOURNAVX in the NOPAIN Act separate payment list, with a retroactive payment date of January 23, 2026. Addition to the NOPAIN list provides a separate payment for non-opioid medicines such as JOURNAVX in the hospital outpatient and ambulatory surgical center settings.
- Vertex has reached an agreement with a major pharmacy benefit manager for Medicare Part D coverage for JOURNAVX, effective May 1. The agreement adds approximately 10 million lives covered under Part D. Twenty-two states now provide coverage for JOURNAVX via Medicaid. In total, approximately 240 million individuals now have reimbursed access to JOURNAVX across a wide range of commercial and government payers.
Select R&D Pipeline Programs
Cystic Fibrosis
- Consistent with its commitment to serial innovation and bringing as many patients as possible to normal levels of CFTR function, Vertex is evaluating VX-828, the first of the next-generation 3.0 CFTR corrector class, in a proof-of-concept study in people with CF. Vertex is on track to complete dosing in this study in the first half of 2026 and share results in the second half.
- Vertex is enrolling and dosing first-in-human studies with VX-581 and VX-272, additional next-generation 3.0 CFTR correctors.
- Vertex has ended the Phase 1/2 study of VX-522 after observing persistent tolerability issues in the study. The early termination precludes assessment of efficacy and full safety and prevents further development of the VX-522 program.
Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
- Vertex continues to advance preclinical assets for gentler conditioning for CASGEVY, which could broaden the eligible patient population.
Acute and Peripheral Neuropathic Pain (PNP)
- Vertex is on track to complete enrollment in both Phase 3 studies of suzetrigine in diabetic peripheral neuropathy (DPN), a form of peripheral neuropathic pain (PNP), by the end of 2026.
- Vertex also continues to enroll and dose people with DPN in a Phase 2 study of VX-993.
- Vertex continues to advance preclinical assets that inhibit NaV1.7 for use alone or in combination with a NaV1.8 inhibitor in acute and neuropathic pain.
IgA Nephropathy (IgAN) and Other B Cell-Mediated Diseases
Vertex is developing povetacicept for multiple diseases. Povetacicept is a dual inhibitor of the BAFF and APRIL cytokines, which play key roles in the pathogenesis of multiple B cell-mediated autoimmune diseases. Povetacicept has pipeline-in-a-product potential and represents a potentially best-in-class approach to control B cell activity in IgAN, primary membranous nephropathy (pMN), and generalized myasthenia gravis (gMG).
-
In March, Vertex reported positive Week 36 interim analysis results for the primary and all secondary endpoints in the RAINIER Phase 3 trial of povetacicept in adults with IgAN. Based on these results, Vertex completed the submission of its rolling biologics license application (BLA) to the FDA in March for potential accelerated approval in the
U.S. Vertex is using a Priority Review Voucher and therefore expects the FDA review of povetacicept’s BLA to be expedited to six months from the date of the FDA’s acceptance of the BLA. - Vertex recently completed enrollment in the Phase 2 portion of the Phase 2/3 OLYMPUS pivotal study of povetacicept in people with pMN and initiated the Phase 3 portion. Enrollment and dosing in the trial are ongoing. The FDA has granted Fast Track and Orphan Drug designations for povetacicept in pMN, and the EMA has granted Priority Medicines (PRIME) designation.
- Vertex has initiated a placebo-controlled, Phase 2 dose-ranging proof-of-concept study evaluating povetacicept for the treatment of gMG.
APOL1-Mediated Kidney Disease (AMKD)
Vertex has discovered and advanced multiple oral, small molecule inhibitors of APOL1 function, pioneering a new class of medicines that targets the underlying cause of this genetic kidney disease.
- In the second half of 2025, Vertex completed enrollment in the interim analysis cohort of the AMPLITUDE Phase 2/3 trial of inaxaplin in people with primary AMKD and will conduct the pre-planned interim analysis for potential accelerated approval after this cohort reaches 48 weeks of treatment. Vertex expects to share data from the interim analysis in early 2027. The AMPLITUDE study is on track to complete full enrollment in the second half of 2026.
- Vertex has completed enrollment in the AMPLIFIED Phase 2 study of inaxaplin. AMPLIFIED is a study of people with AMKD with moderate proteinuria, and people with AMKD and Type 2 diabetes — populations not being studied in the AMPLITUDE trial. Vertex is on track to complete dosing and share data from the AMPLIFIED study in the second half of 2026.
Type 1 Diabetes (T1D)
Vertex is evaluating stem cell-derived, fully differentiated islet cell therapies for patients suffering from T1D, with the goal of developing a potential one-time functional cure for this disease.
- Vertex has completed the internal manufacturing analysis for the Phase 1/2/3 study of zimislecel in people with T1D and has resumed dosing in the study. Multiple patients have been treated since the resumption of dosing. The company expects to provide updated timelines for study completion later this year.
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Vertex is developing small molecule correctors that restore function to polycystin 1 (PC1) protein variants, with the goal of addressing the underlying cause of ADPKD.
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Vertex is enrolling and dosing AGLOW, a Phase 2 study of VX-407 in patients with a subset of variants in the PKD1 gene, which encodes the PC1 protein, estimated to be up to approximately 30,000 (or up to approximately
10% ) of the overall patient population living with ADPKD. - AGLOW is a 24-patient, single-arm, 52-week, Phase 2 proof-of-concept study that will evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV). AGLOW is on track to complete enrollment in the second half of 2026.
Myotonic Dystrophy Type 1 (DM1)
Vertex is evaluating multiple approaches that target the underlying cause of DM1. Vertex’s lead approach, VX-670, is an oligonucleotide linked to a cyclic peptide, which holds the potential to promote effective delivery into cells and address the causal biology of DM1.
- Vertex continues to enroll and dose the MAD portion of the GALILEO global Phase 1/2 clinical trial of VX-670 in people with DM1; the study is assessing both safety and efficacy. Vertex is on track to complete enrollment and dosing in the trial and share results in the second half of 2026.
Additional Earlier Stage R&D Programs
Consistent with its overall strategy, Vertex takes a serial innovation approach to all of its programs, with additional assets or approaches across its portfolio.
Non-GAAP Financial Measures
In this press release, Vertex's financial results and financial guidance are provided in accordance with accounting principles generally accepted in
The company provides guidance regarding combined R&D, AIPR&D and SG&A expenses and effective tax rate on a non-GAAP basis. Unless otherwise noted, the guidance regarding combined R&D, AIPR&D and SG&A expenses does not include estimates associated with any potential future business development transactions, including collaborations, asset acquisitions and/or licensing of third-party intellectual property rights. The company does not provide guidance regarding its GAAP effective tax rate because it is unable to forecast with reasonable certainty the impact of excess tax benefits related to stock-based compensation and the possibility of certain discrete items, which could be material.
Vertex Pharmaceuticals Incorporated Consolidated Statements of Income (unaudited, in millions, except per share amounts) |
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Three Months Ended March 31, |
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|
2026 |
|
|
2025 |
|
|
Revenues: |
|
|
|
|||
Product revenues, net |
$ |
2,986.9 |
|
$ |
2,760.2 |
|
Other revenues |
|
— |
|
|
10.0 |
|
Total revenues |
|
2,986.9 |
|
|
2,770.2 |
|
Costs and expenses: |
|
|
|
|||
Cost of sales |
|
392.8 |
|
|
363.0 |
|
Research and development expenses |
|
961.6 |
|
|
979.7 |
|
Acquired in-process research and development expenses |
|
0.5 |
|
|
19.8 |
|
Selling, general and administrative expenses |
|
493.7 |
|
|
396.4 |
|
Intangible asset impairment charge |
|
— |
|
|
379.0 |
|
Change in fair value of contingent consideration |
|
0.2 |
|
|
2.2 |
|
Total costs and expenses |
|
1,848.8 |
|
|
2,140.1 |
|
Income from operations |
|
1,138.1 |
|
|
630.1 |
|
Interest income, net |
|
114.8 |
|
|
117.9 |
|
Other expense, net |
|
— |
|
|
(17.6 |
) |
Income before provision for income taxes |
|
1,252.9 |
|
|
730.4 |
|
Provision for income taxes |
|
221.5 |
|
|
84.1 |
|
Net income |
$ |
1,031.4 |
|
$ |
646.3 |
|
|
|
|
|
|||
Net income per common share: |
|
|
|
|||
Basic |
$ |
4.06 |
|
$ |
2.52 |
|
Diluted |
$ |
4.02 |
|
$ |
2.49 |
|
Shares used in per share calculations: |
|
|
|
|||
Basic |
|
254.1 |
|
|
256.9 |
|
Diluted |
|
256.3 |
|
|
259.5 |
|
Vertex Pharmaceuticals Incorporated Total Revenues (unaudited, in millions) |
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|
Three Months Ended March 31, |
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|
2026 |
|
2025 |
||
TRIKAFTA/KAFTRIO |
$ |
2,354.7 |
|
$ |
2,535.5 |
ALYFTREK |
|
424.4 |
|
|
53.9 |
Other CF product revenues (1) |
|
135.9 |
|
|
155.3 |
Total CF product revenues, net |
|
2,915.0 |
|
|
2,744.7 |
CASGEVY |
|
42.9 |
|
|
14.2 |
JOURNAVX |
|
29.0 |
|
|
1.3 |
Product revenues, net |
|
2,986.9 |
|
|
2,760.2 |
Other revenues |
|
— |
|
|
10.0 |
Total revenues |
$ |
2,986.9 |
|
$ |
2,770.2 |
|
|
|
|
||
1: Includes KALYDECO, ORKAMBI, and SYMDEKO/SYMKEVI |
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Vertex Pharmaceuticals Incorporated Reconciliation of GAAP to Non-GAAP Financial Information (unaudited, in millions, except percentages) |
|||||||
|
Three Months Ended March 31, |
||||||
|
|
2026 |
|
|
|
2025 |
|
GAAP cost of sales |
$ |
392.8 |
|
|
$ |
363.0 |
|
Stock-based compensation expense |
|
(3.2 |
) |
|
|
(2.6 |
) |
Intangible asset amortization expense |
|
(5.0 |
) |
|
|
(5.0 |
) |
Non-GAAP cost of sales |
$ |
384.6 |
|
|
$ |
355.4 |
|
|
|
|
|
||||
GAAP research and development expenses |
$ |
961.6 |
|
|
$ |
979.7 |
|
Stock-based compensation expense |
|
(101.7 |
) |
|
|
(100.1 |
) |
Intangible asset amortization expense |
|
(0.6 |
) |
|
|
(0.6 |
) |
Non-GAAP research and development expenses |
$ |
859.3 |
|
|
$ |
879.0 |
|
|
|
|
|
||||
Acquired in-process research and development expenses |
$ |
0.5 |
|
|
$ |
19.8 |
|
|
|
|
|
||||
GAAP selling, general and administrative expenses |
$ |
493.7 |
|
|
$ |
396.4 |
|
Stock-based compensation expense |
|
(61.5 |
) |
|
|
(63.4 |
) |
Non-GAAP selling, general and administrative expenses |
$ |
432.2 |
|
|
$ |
333.0 |
|
|
|
|
|
||||
Combined non-GAAP R&D, AIPR&D and SG&A expenses |
$ |
1,292.0 |
|
|
$ |
1,231.8 |
|
|
|
|
|
||||
GAAP other expense, net |
$ |
— |
|
|
$ |
(17.6 |
) |
Decrease in fair value of strategic investments |
|
2.0 |
|
|
|
15.0 |
|
Non-GAAP other income (expense), net |
$ |
2.0 |
|
|
$ |
(2.6 |
) |
|
|
|
|
||||
GAAP provision for income taxes |
$ |
221.5 |
|
|
$ |
84.1 |
|
Tax adjustments (2) |
|
58.6 |
|
|
|
160.1 |
|
Non-GAAP provision for income taxes |
$ |
280.1 |
|
|
$ |
244.2 |
|
|
|
|
|
||||
GAAP effective tax rate |
|
17.7 |
% |
|
|
11.5 |
% |
Non-GAAP effective tax rate |
|
19.6 |
% |
|
|
18.8 |
% |
Vertex Pharmaceuticals Incorporated Reconciliation of GAAP to Non-GAAP Financial Information (continued) (unaudited, in millions, except per share amounts) |
|||||||
|
Three Months Ended March 31, |
||||||
|
|
2026 |
|
|
|
2025 |
|
GAAP operating income |
$ |
1,138.1 |
|
|
$ |
630.1 |
|
Stock-based compensation expense |
|
166.4 |
|
|
|
166.1 |
|
Intangible asset impairment charge |
|
— |
|
|
|
379.0 |
|
Intangible asset amortization expense |
|
5.6 |
|
|
|
5.6 |
|
Increase in fair value of contingent consideration |
|
0.2 |
|
|
|
2.2 |
|
Non-GAAP operating income |
$ |
1,310.3 |
|
|
$ |
1,183.0 |
|
|
|
|
|
||||
GAAP net income |
$ |
1,031.4 |
|
|
$ |
646.3 |
|
|
|
|
|
||||
Stock-based compensation expense |
|
166.4 |
|
|
|
166.1 |
|
Intangible asset impairment charge |
|
— |
|
|
|
379.0 |
|
Intangible asset amortization expense |
|
5.6 |
|
|
|
5.6 |
|
Decrease in fair value of strategic investments |
|
2.0 |
|
|
|
15.0 |
|
Increase in fair value of contingent consideration |
|
0.2 |
|
|
|
2.2 |
|
Total non-GAAP adjustments to pre-tax income |
|
174.2 |
|
|
|
567.9 |
|
Tax adjustments (2) |
|
(58.6 |
) |
|
|
(160.1 |
) |
Non-GAAP net income |
$ |
1,147.0 |
|
|
$ |
1,054.1 |
|
|
|
|
|
||||
Net income per diluted common share: |
|
|
|
||||
GAAP |
$ |
4.02 |
|
|
$ |
2.49 |
|
Non-GAAP |
$ |
4.47 |
|
|
$ |
4.06 |
|
Shares used in diluted per share calculations: |
|
|
|
||||
GAAP and Non-GAAP |
|
256.3 |
|
|
|
259.5 |
|
2: In the three months ended March 31, 2026 and 2025, “Tax adjustments” included the estimated income taxes related to non-GAAP adjustments to the company's pre-tax income and excess tax benefits related to stock-based compensation. |
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Vertex Pharmaceuticals Incorporated Condensed Consolidated Balance Sheets (unaudited, in millions) |
|||||
|
March 31, 2026 |
|
December 31, 2025 |
||
Assets |
|
|
|
||
Cash, cash equivalents and marketable securities |
$ |
7,246.7 |
|
$ |
6,608.1 |
Accounts receivable, net |
|
1,996.1 |
|
|
2,052.8 |
Inventories |
|
1,766.7 |
|
|
1,686.8 |
Prepaid expenses and other current assets |
|
720.8 |
|
|
853.3 |
Total current assets |
|
11,730.3 |
|
|
11,201.0 |
Property and equipment, net |
|
1,608.4 |
|
|
1,520.3 |
Goodwill and other intangible assets, net |
|
1,506.5 |
|
|
1,512.2 |
Deferred tax assets |
|
2,947.8 |
|
|
2,897.9 |
Operating lease assets |
|
1,685.1 |
|
|
1,562.7 |
Long-term marketable securities |
|
5,749.9 |
|
|
5,712.3 |
Other long-term assets |
|
1,256.4 |
|
|
1,236.6 |
Total assets |
$ |
26,484.4 |
|
$ |
25,643.0 |
|
|
|
|
||
Liabilities and Shareholders' Equity |
|
|
|
||
Accounts payable and accrued expenses |
$ |
3,473.5 |
|
$ |
3,432.9 |
Other current liabilities |
|
407.1 |
|
|
428.3 |
Total current liabilities |
|
3,880.6 |
|
|
3,861.2 |
Long-term operating lease liabilities |
|
1,986.5 |
|
|
1,846.5 |
Other long-term liabilities |
|
1,255.4 |
|
|
1,269.5 |
Shareholders' equity |
|
19,361.9 |
|
|
18,665.8 |
Total liabilities and shareholders' equity |
$ |
26,484.4 |
|
$ |
25,643.0 |
|
|
|
|
||
Common shares outstanding |
|
254.2 |
|
|
254.0 |
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, generalized myasthenia gravis, and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements that are subject to risks, uncertainties and other factors. All statements other than statements of historical fact are statements that could be deemed forward-looking statements, including all statements regarding the intent, belief, or current expectation of Vertex and members of the Vertex senior management team. Forward-looking statements are not purely historical and may be accompanied by words such as “anticipates,” “may,” “forecasts,” “expects,” “intends,” “plans,” “potentially,” “believes,” “seeks,” “estimates,” and other words and terms of similar meaning. Such statements include, without limitation, Dr. Kewalramani's statements in this press release, the information provided regarding future financial performance and operations, the section captioned “Full Year 2026 Financial Guidance” and statements regarding (i) expectations for
Conference Call and Webcast
The company will host a conference call and webcast at 4:30 p.m. ET. To access the call, please dial (833) 630-2124 (
The conference call will be webcast live and a link to the webcast can be accessed through Vertex's website at www.vrtx.com in the "Investors" section. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company's website.
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Vertex Pharmaceuticals Incorporated
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or
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or
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Source: Vertex Pharmaceuticals Incorporated