Vertex Pharmaceuticals Incorporated (VRTX): Business Model Canvas [Dec-2025 Updated]

You're digging into the engine room of one of biotech's biggest success stories, and honestly, the Business Model Canvas for Vertex Pharmaceuticals Incorporated as of late 2025 tells a fascinating story: it's all about using the massive, reliable cash flow from their cystic fibrosis (CF) franchise-projected to hit between $11.9 to $12.0 billion in revenue for FY 2025-to aggressively fund a true diversification play. This isn't just about new CF drugs; they are pouring R&D dollars, guided around $5.0 to $5.1 billion for 2025, into transformative areas like the functional cure Casgevy for blood disorders and the acute pain treatment Journavx, all while sitting on a war chest of $12.0 billion in cash by Q3. If you want to see exactly how this powerhouse balances its dominant core business with its high-stakes expansion bets, you need to see the nine building blocks laid out below.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Key Partnerships

You're looking at the core relationships that fuel Vertex Pharmaceuticals Incorporated's growth beyond its established cystic fibrosis (CF) franchise. These alliances are critical for diversifying revenue and filling the pipeline, so let's look at the hard numbers defining these deals as of late 2025.

CRISPR Therapeutics for global Casgevy development and commercialization

The co-development and commercialization agreement for CASGEVY is structured with Vertex leading global development, manufacturing, and sales. This arrangement dictates a worldwide cost and profit split of 60/40 in favor of Vertex Pharmaceuticals Incorporated.

The financial performance of CASGEVY in 2025 shows accelerating adoption, though the ramp-up has been gradual. Vertex Pharmaceuticals Incorporated expects to record over $100 million in total CASGEVY revenue for the full year 2025, with significant growth projected into 2026.

Here's a look at the reported revenue and patient metrics for CASGEVY through the third quarter of 2025:

Vertex Pharmaceuticals Incorporated also plans to begin manufacturing CASGEVY in its Portsmouth, NH facility during the second half of 2025.

Moderna for the development of VX-522, a CFTR mRNA therapeutic

VX-522 is designed for the estimated >5,000 people with CF who cannot benefit from existing CFTR modulators. Vertex Pharmaceuticals Incorporated entered the initial research collaboration with Moderna in 2020, paying $75 million for a three-year term that yielded VX-522.

The clinical program has seen some turbulence; the multiple ascending dose (MAD) portion of the Phase 1/2 study was temporarily paused in May 2025 due to a tolerability issue. Data from the single ascending dose (SAD) portion was expected in the first half of 2025. The study itself began dosing on December 19, 2022.

Alpine Immune Sciences acquisition, integrating povetacicept for kidney disease

Vertex Pharmaceuticals Incorporated agreed to acquire Alpine Immune Sciences in a deal valued at $4.9 billion in cash. The per-share price was $65 cash. Povetacicept is targeting IgA nephropathy (IgAN), a condition affecting an estimated 130,000 people in the U.S.

The integration is already impacting guidance, as Vertex Pharmaceuticals Incorporated raised its expense guidance to account for the acceleration of the povetacicept pipeline program. The company expects to initiate a rolling BLA (Biologics License Application) filing for povetacicept for IgAN by the end of 2025.

Global network of over 75 Authorized Treatment Centers (ATCs) for Casgevy

Building out the specialized infrastructure for CASGEVY administration is a key operational focus. The number of activated ATCs has been steadily increasing:

• As of the end of 2024, more than 50 ATCs were activated globally.
• By May 1, 2025, this number grew to more than 65 ATCs, against a goal of 75 globally.
• As of the Q2 2025 earnings call, more than 75 ATCs were activated across all approved regions.

Regarding patient throughput, as of June 2025-end, about 115 patients had completed their first cell collection.

Academic research institutions for early-stage drug discovery and clinical trials

Vertex Pharmaceuticals Incorporated maintains relationships with academic research institutions. These partnerships support early-stage drug discovery and are integral to running clinical trials. No specific financial or statistical data regarding the monetary value or number of these specific agreements is available in the latest reports.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Key Activities

You're managing a company with a dominant franchise that's successfully pivoting into new, complex therapeutic areas; the key activities have to reflect both defense of the core and aggressive expansion. For Vertex Pharmaceuticals Incorporated, this means a dual focus on maximizing the existing small molecule base while executing on cell/gene therapy launches.

Research and development (R&D) for non-CF pipeline programs like povetacicept

Vertex Pharmaceuticals Incorporated is heavily investing in its non-CF pipeline, which is a critical activity for long-term growth beyond its core business. The company is advancing povetacicept, a dual inhibitor of the BAFF and APRIL pathways, for indications like immunoglobulin A nephropathy (IgAN) and primary membranous nephropathy (pMN). This program was significantly bolstered by the $4.9 billion takeover of Alpine Immune Sciences last year. To support this acceleration, Vertex revised its full-year 2025 guidance for combined GAAP R&D, Acquired IPR&D (AIPR&D), and Selling, General & Administrative (SG&A) expenses to be approximately $5.65 to $5.8 billion. The non-GAAP expense guidance for the same categories was revised to $5.0 to $5.1 billion. Enrollment in the global Phase 3 RAINIER study of povetacicept in IgAN is now complete. Vertex is on track to submit the first module of the povetacicept IgAN Biologics License Application (BLA) to the FDA by the end of 2025.

• Enrollment complete: povetacicept Phase 3 IgAN trial.
• FDA BLA submission planned for IgAN by end of 2025.
• Pivotal Phase II/III study underway for pMN.

Global commercialization and life-cycle management of the CF portfolio (e.g., Alyftrek launch)

Maintaining and expanding the Cystic Fibrosis (CF) franchise remains a foundational activity, driving the majority of the company's revenue. Vertex Pharmaceuticals Incorporated predicts total full-year 2025 revenue to be in the range of $11.9 billion to $12.0 billion. The third quarter of 2025 saw total revenue hit $3.08 billion, marking an 11% increase compared to Q3 2024. The CF franchise was responsible for $2.9 billion of that Q3 revenue. In 2023, the CF franchise contributed over USD 9.3 billion, representing 94% of the total company revenue that year. The launch of ALYFTREK contributed to the revenue increase seen in the first quarter of 2025.

Executing the complex launch and reimbursement strategy for Casgevy

The launch of Casgevy (exagamglogene autotemcel), the first CRISPR/Cas9 gene-edited therapy, requires a specialized commercial and access strategy. Vertex expects over $100 million in Casgevy revenues for the full year 2025. In the third quarter of 2025, Casgevy generated $16.9 million in sales. Cumulatively, for the first nine months of 2025, Casgevy revenues reached $61.5 million. As of September 30, 2025, approximately 165 people globally had initiated cell collection for Casgevy, with 39 people having received infusions. This launch is complicated by the need for specialized treatment centers and managing the logistics of a cell/gene therapy. Still, the progress is defintely notable.

Manufacturing and supply chain for small molecule and cell/gene therapies

Vertex Pharmaceuticals Incorporated is scaling its manufacturing footprint to support both its small molecule dominance and the emerging cell/gene therapy portfolio. The company is expanding its Boston footprint, which is expected to grow to approximately 1.8M square feet by 2026, representing a ~50% increase from 2019 levels. Furthermore, construction is underway on another building in Leiden, which is expected to be finished by 2026. The Providence, Rhode Island R&D sites include existing facilities of 50,000-square-foot and 22,000-square-foot, with plans to add new manufacturing lines and upgrade utilities to expand clean room capabilities for cell and genetic therapies. For cell therapy production, Vertex also leverages external partnerships, such as the one with Lonza.

Securing reimbursement agreements with national health systems and payers

A key activity following regulatory approval for specialized therapies like Casgevy is securing access through reimbursement. Vertex announced a reimbursement agreement with the Italian Medicines Agency (AIFA) in September 2025 for eligible patients with transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD) to access Casgevy. This agreement was a turning point for eligible people in Italy. The company continues to work with reimbursement authorities globally to establish sustainable access for additional eligible patients.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Key Resources

You're looking at the core assets that make Vertex Pharmaceuticals Incorporated a powerhouse in the specialty pharma space. These aren't just ideas; they are tangible, high-value resources underpinning their entire operation, especially as they push beyond their core cystic fibrosis (CF) franchise.

Proprietary intellectual property (IP) covering CFTR modulator therapies is the bedrock. Vertex has successfully built a moat around CF treatment, holding approvals for eight medicines, with seven dedicated to CF. Their latest CFTR modulator, ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor), demonstrated superiority to TRIKAFTA in lowering sweat chloride (SwCl) in Phase 3 trials, suggesting even greater potential for patient benefit. The IP strength is evidenced by the breadth of coverage: TRIKAFTA is now approved for patients with a total of 272 CFTR mutations in the U.S., and the EU label for KAFTRIO was expanded to include patients with at least one non-class I mutation in the CFTR gene. Furthermore, they are developing VX-522, a CFTR mRNA therapy via lipid nanoparticles, aimed at the estimated 5,000 people with CF who do not produce any CFTR protein responsive to current modulators. This continuous innovation solidifies their IP position.

The financial muscle supporting this pipeline is substantial. As of September 30, 2025, Vertex Pharmaceuticals Incorporated held $12.0 billion in cash, cash equivalents, and total marketable securities. This war chest grew from $11.2 billion at the end of 2024. This liquidity is crucial, especially as the company guides for full-year 2025 total revenue between $11.9 billion and $12.0 billion. Honestly, that cash position gives them incredible flexibility for R&D acceleration and strategic moves.

The talent pool is highly specialized, focusing on areas where they possess a deep understanding of the underlying human biology. This includes expertise in small molecule drug discovery, which is evident in their pain and kidney disease programs. For instance, they have discovered and advanced multiple oral, small molecule inhibitors targeting the underlying cause of APOL1-Mediated Kidney Disease (AMKD). They are also heavily invested in advanced modalities:

• Pioneering stem cell-derived, fully differentiated islet cell therapies (Zimislecel) for Type 1 Diabetes (T1D).
• Researching in vivo gene editing approaches for Transfusion-Dependent Beta Thalassemia (TDT).
• Developing small molecule correctors for Autosomal Dominant Polycystic Kidney Disease (ADPKD).

The planned transition of the Chief Scientific Officer role, with Mark Bunnage taking over in February 2026, shows a commitment to continuity in this scientific leadership.

Global regulatory expertise for ultra-rare and specialty disease approvals is demonstrated by their successful navigation of complex regulatory pathways across multiple novel mechanisms of action. You can see this in their recent commercial successes and pipeline progression:

Finally, the portfolio of marketed products provides the immediate revenue stream funding this expansion. As of Q3 2025, the key revenue drivers outside of the established CF portfolio include:

• CASGEVY: Generated $17 million in revenue in Q3 2025.
• JOURNAVX: Contributed $20 million in revenue in Q3 2025.

The company is executing on its diversification plan, with all three new launches-CASGEVY, ALYFTREK, and JOURNAVX-contributing to the Q3 2025 total revenue of $3.08 billion. Finance: draft 13-week cash view by Friday.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Value Propositions

You're looking at the core reasons why Vertex Pharmaceuticals Incorporated commands its market position as of late 2025. It's all about targeting the root cause of serious diseases with transformative medicines, moving beyond just managing symptoms. The value propositions are concrete, backed by recent financial and clinical performance.

For Cystic Fibrosis (CF), the value proposition centers on market leadership and next-generation efficacy. Vertex CF medicines are currently treating over 75,000 people with CF in more than 60 countries on six continents. This represents approximately 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. The newest offering, Alyftrek, is key here, offering a once-daily dosing profile compared to the twice-daily regimen of its predecessor, Trikafta.

The shift to Alyftrek is priced at a wholesale acquisition cost (WAC) of about $370,269 per year, which is a roughly 7% premium over Trikafta's approximate $346,000 annual WAC. The early adoption shows in the numbers; Alyftrek contributed to a 15% increase in U.S. revenue in Q3 2025, with Q1 2025 revenue reported at $171 million. Honestly, the convenience of once-daily dosing is a massive value driver for patient adherence.

Vertex is also delivering on the promise of a functional cure for Sickle Cell Disease (SCD) and Transplant-Dependent $\beta$-Thalassemia (TDT) with Casgevy. The clinical data is compelling: in SCD patients with at least 16 months of follow-up, 43 out of 45 evaluable patients (95.6%) were free from vaso-occlusive crises (VOCs) for at least 12 consecutive months (VF12). The global uptake is building, with 29 patients having received infusions of CASGEVY as of June 30th, 2025. The addressable market is substantial, with an estimated 35,000 potential patients in the U.S. and Europe, plus another 23,000 eligible patients in Saudi Arabia and Bahrain.

The acute pain franchise is now diversified with Journavx (suzetrigine), the first new class of acute pain medication in over 20 years. This non-opioid treatment targets the 80 million people in America prescribed medicine for moderate-to-severe acute pain annually. Journavx is already showing commercial traction, reporting $19.6 million in revenue in Q3 2025, with prescriptions surpassing 300,000 and securing access for over 170 million covered lives by the end of that quarter.

Here's a quick look at the key metrics for the commercial launches as of late 2025:

Also, Vertex is positioning itself for the next wave of growth with transformative pipeline assets, particularly in kidney disease, where causal human biology is well-understood. The company is on track to submit the first module of the Biologics License Application (BLA) for povetacicept in IgAN to the FDA by the end of 2025, following completion of full enrollment in the Phase 3 RAINIER trial. For APOL1-mediated kidney disease (AMKD), enrollment for the interim analysis cohort of the pivotal AMPLITUDE trial is complete, setting up a potential accelerated approval filing if the data supports it.

The pipeline value propositions include:

• Potential transformative therapies for IgAN (povetacicept) and AMKD (inaxaplin).
• Povetacicept in IgAN received Breakthrough Therapy Designation (BTD) from the U.S. FDA.
• Phase 3 IgAN trial full enrollment complete as of September 30, 2025.
• Inaxaplin for AMKD has completed enrollment for the interim analysis cohort, with results expected to support a potential U.S. accelerated approval filing.

Finance: review the Q3 2025 revenue contribution breakdown across the three launched products for the 2026 budget by next Wednesday.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Customer Relationships

You're looking at how Vertex Pharmaceuticals Incorporated (VRTX) manages relationships with the highly specialized and often vulnerable patient populations it serves, especially with its newer, complex therapies. This isn't about mass-market sales; it's about intensive, coordinated care.

High-touch, specialized patient support programs for complex therapies like Casgevy.

For its gene-edited therapy, CASGEVY, the relationship starts with a highly structured, multi-step process requiring specialized infrastructure. Vertex Connects is the dedicated program for patients prescribed this gene therapy and their loved ones. Care Managers are available Monday through Friday, from 8 AM to 8 PM ET, to guide patients through the treatment journey, which involves cell collection, manufacturing, conditioning medicine, and infusion.

The physical infrastructure supporting this relationship is the network of Authorized Treatment Centers (ATCs). Vertex has met its goal of activating more than 75 ATCs globally. As of September 30, 2025, approximately 165 people with SCD or TDT had their first cell collection globally, and 39 people had received infusions. In the U.S., through the end of September 2025, 25 ATCs had initiated more than 5 patients each.

Here's a snapshot of the CASGEVY treatment initiation progress as of late 2025:

Direct engagement with government and private payers for pricing and access agreements.

Securing access is a critical relationship point, especially for high-cost, transformative medicines. For CASGEVY, Vertex has secured reimbursement agreements for eligible patients in 10 countries as of mid-2025, with recent agreements in places like Northern Ireland, Scotland, and Denmark. Italy, which has the largest TDT population in Europe, signed a reimbursement agreement for TDT and SCD in Q3 2025.

For the acute pain medicine JOURNAVX, market access in the U.S. is heavily managed through payer relationships. As of mid-July 2025, nearly 150 million individuals had covered access across commercial and government payers, which is almost half of U.S. covered lives. This access includes formal coverage agreements with two of the three large national Pharmacy Benefit Managers (PBMs). Furthermore, unrestricted access (no prior authorization or step edits) was available within 16 state Medicaid plans as of mid-July 2025.

Dedicated medical affairs teams supporting specialty physicians and ATCs.

The relationship with specialty physicians centers on the highly specialized nature of the treatments, particularly the cell therapy centers (ATCs). Vertex Pharmaceuticals has over 6,000 employees worldwide, with approximately 5,000 in the U.S. While a specific number for the Medical Affairs team isn't public, the focus is clearly on supporting the centers administering the therapy. The Vertex Physician Investigator Career Development Program highlights an investment in physician-scientists, suggesting a deep, long-term relationship with the medical community to advance science and treatment protocols.

Long-term patient adherence programs for chronic CF therapies.

For the chronic Cystic Fibrosis (CF) patient base, the relationship shifts to ensuring long-term adherence to daily, often burdensome, medication regimens. Vertex CFTR modulators can treat nearly 95 percent of all people living with CF in core markets. Today, Vertex medicines treat over 75,000 people with CF in more than 60 countries on six continents, representing approximately 2/3 of diagnosed, eligible CF patients. In the U.S., access is broad, with more than 99% of eligible patients covered through public and private insurance.

The challenge remains adherence, as CF patients can take 8-10 medications daily, with regimens taking up to 2-3 hours. Adherence rates among people with CF can range from approximately 35% to 75% depending on the measurement method and patient group. Vertex supports care teams by providing strategies to address psychosocial factors, reinforce benefits, and encourage the use of technology for adherence. That's a lot of daily effort for patients.

Key metrics for the established CF customer base:

• CF patients treated globally: Over 75,000.
• Global countries treating patients: Over 60.
• US patient insurance coverage: Over 99%.
• Reported adherence range: 35% to 75%.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Channels

You're looking at how Vertex Pharmaceuticals Incorporated gets its transformative medicines-from pills for cystic fibrosis (CF) to cutting-edge cell therapies-into the hands of the patients who need them. The channels are complex because the products are so different, so let's break down the logistics for late 2025.

Specialty pharmacies and distributors for oral CF and pain medicines

For Vertex Pharmaceuticals Incorporated's established oral CF therapies like TRIKAFTA/KAFTRIO and the newer ALYFTREK, distribution relies on established pharmaceutical supply chains. TRIKAFTA/KAFTRIO is approved for patients with a total of 272 CFTR mutations as of January 2025, and ALYFTREK is approved for patients with a total of 303 mutations as of January 2025. The newer non-opioid pain medicine, JOURNAVX, which received FDA approval in January 2025, also flows through these established networks. While the exact number of specialty pharmacy partners isn't public, these oral products leverage broad distribution channels, including those that handle specialty drugs, to reach retail pharmacies and health systems. The company is expecting total revenue for the full year 2025 to be between $11.9 to $12.0 billion, with these oral launches contributing to that figure.

Authorized Treatment Centers (ATCs) for Casgevy cell collection and infusion

The channel for CASGEVY, the gene-editing therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), is highly specialized. This therapy requires an intensive, multi-step process managed exclusively through Authorized Treatment Centers (ATCs). Vertex Pharmaceuticals Incorporated achieved its goal of activating 75 ATCs globally through September 30, 2025. This infrastructure is critical for managing the complex logistics of cell collection, conditioning, and infusion. As of September 30, 2025, approximately 165 people with SCD or TDT had their first cell collection, and 39 people had received CASGEVY infusions across these centers. The company negotiated a first-of-its-kind, voluntary agreement with the Centers for Medicare & Medicaid Services (CMS) in the U.S. to ensure broad access through state Medicaid programs.

Here's a snapshot of the CASGEVY channel progress as of the end of the third quarter of 2025:

Direct sales force targeting pulmonologists, hematologists, and pain specialists

Vertex Pharmaceuticals Incorporated deploys specialized commercial teams to support the adoption of its targeted therapies. For the CF portfolio, the sales force engages directly with pulmonologists who manage the patient base for TRIKAFTA/KAFTRIO and ALYFTREK. With the launch of JOURNAVX, a direct sales presence targets pain specialists. Hematologists and transplant specialists are the key prescribers and coordinators for the CASGEVY treatment journey within the ATC network. While specific headcount isn't reported, the focus is on educating these high-specialty physicians on the science and administration protocols for the newer products. The company's Q3 2025 total revenue reached $3.08 billion, reflecting the commercial execution across these specialist groups.

Global regulatory bodies (FDA, EMA) for market access and label expansion

Regulatory bodies are a crucial channel for market access and expanding the patient population Vertex Pharmaceuticals Incorporated can serve. The company actively engages with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). For CASGEVY, approvals secured include the U.S., the EU, Great Britain, Canada, Switzerland, and several Middle Eastern nations. For its pipeline, the investigational T1D therapy, zimislecel, has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA, plus Priority Medicines (PRIME) designation from the EMA. Vertex is on track to submit the first module of the povetacicept IgAN Biologics License Application (BLA) to the FDA before the end of 2025, aiming for potential U.S. accelerated approval. These regulatory milestones directly enable commercial channel activation.

Key regulatory channel achievements include:

• FDA approval for JOURNAVX in January 2025.
• FDA approval for ALYFTREK in December 2024.
• EMA authorization to initiate a Phase 2 Clinical Trial of SRSD107 in 2025.
• FDA granted Priority Review for suzetrigine (JOURNAVX) with a PDUFA target action date of January 30, 2025.

Finance: draft 13-week cash view by Friday.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Customer Segments

You're looking at the core patient populations that Vertex Pharmaceuticals Incorporated is serving or targeting as of late 2025. This is where the revenue engine is, and where the future pipeline is aimed. It's a focused approach, hitting specific, high-unmet-need genetic diseases first.

Cystic Fibrosis (CF) patients eligible for CFTR modulators (the core market)

This remains the foundation of Vertex Pharmaceuticals Incorporated's commercial success. The company has seen massive expansion here, moving from a small patient base to treating a significant majority of the addressable population globally with its CFTR modulators like TRIKAFTA and the newly approved ALYFTREK.

As of late 2025, Vertex medicines are treating more that 75,000 patients across more than 60 countries on six continents. This represents approximately 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. The total estimated population living with cystic fibrosis is around 109,000 people.

The market focus in the U.S., Europe, Australia, and Canada covers approximately 94,000 diagnosed individuals. The company continues to expand the reach of its existing portfolio:

• ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor) is approved for patients with at least one F508del mutation or another mutation responsive to the drug, covering a total of 303 mutations as of December 20, 2024.
• TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is approved for patients with a total of 272 CFTR mutations as of December 20, 2024.
• In August 2025, Vertex announced FDA approval for VX-548, a next-generation CFTR modulator for patients with rare mutations, expected to expand eligibility to thousands previously lacking options.
• The population that cannot benefit from current CFTR modulators is estimated at over 5,000 people, the target for the investigational VX-522 mRNA therapeutic.

Access in the U.S. is nearly universal for eligible patients, with more than 99% having coverage through public and private insurance. The strong performance of the CF portfolio, including ALYFTREK, was a primary driver of Vertex Pharmaceuticals Incorporated's Q3 2025 total revenue of $3.08 billion, which was an 11% increase year-over-year.

Patients with Sickle Cell Disease (SCD) and Transfusion-Dependent Beta-Thalassemia (TDT)

This segment is being addressed with the transformative gene-edited therapy, CASGEVY (exagamglogene autotemcel). This patient group has historically faced severe morbidity, with U.S. lifetime healthcare costs for managing recurrent VOCs estimated between $4 and $6 million per patient.

CASGEVY is currently approved for eligible people ages 12 years and older in the U.S., Great Britain, the EU, and several other regions. Data from pivotal trials show durable benefits:

Vertex is focused on expanding this customer base to younger patients, with plans to initiate global regulatory submissions for children ages 5-11 years in the first half of 2026. Early contributions from CASGEVY are now factored into the refined full-year 2025 revenue guidance of $11.9 to $12.0 billion.

Patients experiencing acute pain (post-surgical, etc.) for Journavx

This represents Vertex Pharmaceuticals Incorporated's newest commercial segment, following the FDA approval of JOURNAVX (suzetrigine) on January 30, 2025. The target market is substantial, as more than 80 million people in America are prescribed medicine for moderate-to-severe acute pain each year. Of those, about 40 million are prescribed opioids, with roughly 85,000 developing opioid use disorder annually.

JOURNAVX is positioned as a first-in-class, non-opioid treatment, which is a significant differentiator. The financial opportunity is large; one analyst models the drug reaching $1 billion in sales by 2028 and peaking at about $4.9 billion in sales in 2031. Early contributions from the U.S. launch of JOURNAVX were noted as a growth driver in Q3 2025.

Patients with IgA Nephropathy (IgAN) and APOL1-mediated kidney disease (future)

These are future, high-unmet-need customer segments where Vertex Pharmaceuticals Incorporated is advancing therapies targeting the underlying cause of disease. IgA Nephropathy (IgAN) affects approximately 300,000 people in the United States and Europe.

Progress in these areas is measured by clinical trial milestones as of late 2025:

• IgAN: The Phase 3 RAINIER trial for povetacicept completed full enrollment in record time, less than 15 months. Vertex is on track to submit the first module of the Biologics License Application (BLA) to the FDA by the end of 2025 for potential U.S. accelerated approval.
• APOL1-mediated kidney disease (AMKD): Enrollment is complete for the interim analysis cohort of the AMPLITUDE global Phase 2/3 trial evaluating inaxaplin. Results are expected next year, 2026, to support a potential U.S. accelerated approval filing.

These pipeline advancements are critical to the company's strategy of serial innovation beyond its current commercial base. Finance: draft 2026 R&D budget allocation for kidney programs by next Wednesday.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Cost Structure

You're looking at where Vertex Pharmaceuticals Incorporated is putting its capital to work to drive growth beyond its core Cystic Fibrosis franchise. The cost structure is heavily weighted toward future innovation and market expansion.

The primary cost driver is the combined Research & Development (R&D), Acquired Intellectual Property Rights & Research & Development (AIPR&D), and Selling, General & Administrative (SG&A) expenses. Vertex Pharmaceuticals Incorporated reiterated its full-year 2025 guidance for these combined non-GAAP expenses to be in the range of $5.0 to $5.1 billion. This figure reflects significant ongoing investment across the board.

A specific component of this guidance is the expected cost related to Acquired IPR&D, which is guided to be approximately $100 million for fiscal year 2025.

The investment in commercialization is substantial, directly supporting the launches of new products. This is evident in the rising SG&A component, which covers the build-out of global commercial capabilities. For instance, the combined non-GAAP R&D, AIPR&D, and SG&A expenses for the third quarter of 2025 reached $1.3 billion, up from $1.1 billion in the third quarter of 2024. This increase was explicitly attributed to increased commercial investment supporting the launch of JOURNAVX in acute pain, alongside R&D investments.

The R&D portion of the cost structure is dedicated to advancing a deep pipeline, with five programs in pivotal development as of late 2025. This late-stage clinical work requires significant outlay for trials, such as the povetacicept Phase 3 IgAN trial which completed full enrollment.

Manufacturing costs are also a key area, particularly for complex modalities. The costs associated with manufacturing for gene and cell therapies, such as CASGEVY, represent a specialized and high-value component of the overall cost of goods sold and operational expenses, though specific manufacturing cost guidance separate from the combined operating expenses is not typically broken out in the same manner.

Here's a look at the trend in the combined operating expenses for the first three quarters of 2025 compared to the prior year:

The focus on commercial execution is also reflected in the SG&A component, which is increasing to support the global rollout of multiple products simultaneously. You can see the commitment to pipeline advancement through the R&D spend, which is necessary to bring those five pivotal programs toward potential filings in 2026.

The key elements driving the cost base are:

• Continued investment in R&D for multiple mid- and late-stage programs.
• Increased commercial investment for JOURNAVX and CASGEVY launches.
• AIPR&D expense guidance of approximately $100 million for the full year 2025.
• Costs associated with five programs in pivotal clinical development.
• Manufacturing scale-up for complex therapies like CASGEVY.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Revenue Streams

You're looking at the core money-makers for Vertex Pharmaceuticals Incorporated as of late 2025. The revenue streams are clearly anchored by the established Cystic Fibrosis (CF) franchise, but the growth story is all about the newer launches.

The total revenue guidance for Vertex Pharmaceuticals Incorporated for the full fiscal year 2025 is set between $11.9 to $12.0 billion. This guidance assumes the continued growth of the CF portfolio, including the global rollout of ALYFTREK, alongside the uptake of CASGEVY and early revenue from JOURNAVX.

Product sales from the Cystic Fibrosis franchise remain the foundation. This franchise includes Trikafta/Kaftrio and the newly launched ALYFTREK, which is Vertex Pharmaceuticals Incorporated's fifth CF medicine. The continued performance of these CF therapies was the primary driver for the 11% increase in total revenue to $3.08 billion in the third quarter of 2025 compared to the third quarter of 2024.

Newer products are diversifying this base. Vertex Pharmaceuticals Incorporated expects over $100 million in total CASGEVY revenues for the full year 2025. Through the first nine months of 2025, CASGEVY recorded sales of $61.5 million. For JOURNAVX (suzetrigine) in acute pain, the company recorded sales of $32.9 million for the first nine months of 2025. In the second quarter of 2025 specifically, JOURNAVX contributed $12 million to revenue.

Collaboration revenue also contributes to the top line, though it is a smaller component relative to product sales. For instance, in the second quarter of 2025, Vertex recorded $21 million in collaboration revenue. The partnership with CRISPR Therapeutics for CASGEVY involves Vertex leading global development and commercialization, splitting program costs and profits worldwide 60:40.

Here's a snapshot of the reported and guided revenue components for 2025, based on the latest available data points:

The company is actively investing in the commercialization of JOURNAVX in acute pain, which is expected to see increased sales in the fourth quarter as prescription volumes rise. Vertex Pharmaceuticals Incorporated is also focused on expanding global momentum for CASGEVY, with significant growth expected in 2026.

You can see the focus is shifting from a single-disease revenue base to a multi-product portfolio supporting the overall guidance.

• Continued growth in CF product demand, including for ALYFTREK.
• Early contributions from the U.S. launch of JOURNAVX.
• Anticipated continued uptake of CASGEVY in multiple regions.
• The CF franchise treats nearly 75% of the 94,000 patients living with CF in the United States, Europe, Canada and Australia.