BioMarin Pharmaceutical Inc. (BMRN): Business Model Canvas [Dec-2025 Updated]

You're looking to dissect the engine behind BioMarin Pharmaceutical Inc.'s success, and honestly, it boils down to mastering the ultra-orphan space. After two decades analyzing pharma, including my time leading analysis at BlackRock, I can tell you their model isn't about volume; it's about delivering durable, life-saving therapies like Voxzogo to tiny patient populations, which is why they are guiding for $3.15 billion to $3.2 billion in total revenue for 2025. They trade high R&D costs and complex manufacturing for premium pricing and deep patient relationships. This is how you build a fortress in rare diseases. Below, we break down the nine essential blocks of their current business strategy, from key resources to their specialized customer channels, so you can see exactly how they capture value.

BioMarin Pharmaceutical Inc. (BMRN) - Canvas Business Model: Key Partnerships

You're looking at the structure that supports BioMarin Pharmaceutical Inc.'s ultra-orphan drug focus as of late 2025. The partnerships are critical for both pipeline expansion and getting those specialized medicines to the few patients who need them.

Global Regulatory Bodies (FDA, EMA) for Product Approvals

Engagement with global regulatory bodies like the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) is a constant for BioMarin Pharmaceutical Inc., especially with pipeline assets moving toward submission. The company is targeting regulatory applications to expand the label of Palynziq to treat adolescents with phenylketonuria (PKU) between the ages of 12 and 17 in both the U.S. and Europe in the 2nd half of 2025. Furthermore, the newly acquired asset, BMN 401, has pivotal Phase 3 data expected in the 1H 2026, with potential regulatory approval targeted for 2027.

Specialty Pharmacies and Distributors for Ultra-Orphan Drug Delivery

Delivering ultra-orphan drugs requires tight control over the supply chain, relying heavily on specialized partners. While BioMarin Pharmaceutical Inc.'s specific contract numbers aren't public, the environment they operate in is massive; the overall specialty drug distribution market is projected to reach $275.36 billion in 2025. The largest players in this space control an estimated 70% of that market. BioMarin Pharmaceutical Inc.'s existing Enzyme Therapies portfolio, which includes five commercial therapies, generated $555 million in revenue in the second quarter of 2025, marking a 15% year-over-year increase.

The reliance on this channel is key to supporting the company's financial footing, which saw total revenues of $825 million in Q2 2025.

Academic Research Institutions for Early-Stage Discovery and Trials

The pipeline advancement shows a reliance on external scientific validation. For instance, the company is advancing BMN 333, its long-acting C-type natriuretic peptide, with early clinical results shared in Q1 2025, and a pivotal Phase 2/3 study planned to start in the 1H 2026. This type of development relies on foundational research often originating from or validated by academic centers.

Inozyme Pharma (Acquired July 2025) for Pipeline Assets like BMN 401

The acquisition of Inozyme Pharma in July 2025 was a major partnership move to bolster the late-stage pipeline. This transaction was an all-cash deal valued at approximately $270 million. The deal was executed at $4.00 per share. This acquisition brought in BMN 401 (formerly INZ-701), an enzyme replacement therapy for ENPP1 Deficiency, a condition affecting an estimated 1,000-2,000 patients globally.

Here are the key financial and asset details surrounding this specific partnership integration:

The company's strong financial position, ending Q2 2025 with approximately $1.94 billion in cash, supported this strategic external innovation move.

BioMarin Pharmaceutical Inc. (BMRN) - Canvas Business Model: Key Activities

You're looking at the core engine of BioMarin Pharmaceutical Inc., the activities that actually turn their science into revenue and patient impact. It's a high-stakes game of discovery, scale-up, and securing payer approval for very expensive, specialized treatments. Here's the breakdown of what BioMarin is actively doing as of late 2025.

Research and development (R&D) of novel genetic therapies

The R&D function is central, though BioMarin Pharmaceutical Inc. has been actively pruning its portfolio to focus resources. Research and Development Expenses for the twelve months ending September 30, 2025, reached $0.903 billion, marking a 15.78% increase year-over-year from the 2024 full-year expense of $0.747 billion. This spending is now concentrated on a streamlined set of high-potential candidates following a strategic portfolio review announced in 2024.

The key activity here is advancing these prioritized programs through clinical stages, with specific milestones targeted for late 2025:

• Advance pipeline candidates including BMN 333, BMN 349, and BMN 351.
• Expect initial clinical data read-outs for BMN 351 for Duchenne Muscular Dystrophy by year-end 2025.
• Completed enrollment in the pivotal hypochondroplasia study for VOXZOGO in April 2025.
• Completed the acquisition of Inozyme on July 1, 2025, adding BMN 401 (formerly INZ-701).

The company is definitely shifting its investment focus, as Q1 2025 saw a decrease in R&D expenses year-over-year due to cost transformation initiatives, and Q2 2025 saw lower R&D spend due to re-prioritization.

Global commercialization of rare disease medicines like Voxzogo

Commercialization is heavily driven by VOXZOGO, the treatment for achondroplasia, which is seeing continued global expansion. Full-year 2025 Total Revenues are guided to be between $3.1 billion and $3.2 billion, with VOXZOGO expected to contribute between $900 million and $935 million of that total. This is up from $735 million in global revenue for the full year 2024.

The commercial footprint is expanding rapidly:

BioMarin Pharmaceutical Inc. is targeting access to VOXZOGO in over 60 countries by 2027.

Manufacturing of complex biologic and enzyme replacement therapies

BioMarin Pharmaceutical Inc. relies on an owned manufacturing approach to maintain quality standards and meet demand for its complex therapies, including enzyme replacement therapies and gene therapy like ROCTAVIAN. The company operates three cGMP (current Good Manufacturing Practices) manufacturing plants.

A major recent activity was the investment to scale up capacity:

• Announced €60 million (US$63 million) investment in December 2024 to expand the Shanbally, Co. Cork, Ireland facility.
• The Shanbally site handles end-to-end manufacturing, from bulk drug substance to final packaging.
• This investment adds a new, four-story laboratory to increase production capacity for approved drugs.

The Novato, California facility previously could support approximately $1.0 billion in revenue.

Managing global reimbursement and market access for high-cost treatments

Securing reimbursement is critical for these high-cost, specialized treatments. For ROCTAVIAN, the gene therapy for severe hemophilia A, BioMarin Pharmaceutical Inc. has strategically focused commercial operations on the U.S., Germany and Italy, the markets where it is both approved and reimbursed.

The company is actively managing the economics of this product:

• Anticipates reducing annual direct ROCTAVIAN expenses to approximately $60 million, beginning in 2025.
• Expects ROCTAVIAN to be profitable by the end of 2025.
• In the pivotal study, 82% of participants remained off prophylaxis at year four.
• Adoption has been slow, with only 5 patients treated as of Q3 2025.

Overall financial health supports these activities; BioMarin Pharmaceutical Inc. generated operating cash flows totaling $728 million year-to-date as of Q3 2025, with total cash and investments at approximately $2.0 billion at the end of that quarter. Finance: draft 13-week cash view by Friday.

BioMarin Pharmaceutical Inc. (BMRN) - Canvas Business Model: Key Resources

Cash and investments of approximately $2.0 billion as of Q3 2025.

The Key Resources supporting BioMarin Pharmaceutical Inc.'s operations include:

• Portfolio of eight commercial therapies for rare genetic diseases.
• Intellectual property (IP) protecting its enzyme and peptide platforms, including over 15 years of scientific research supporting the CNP patent.

The company's physical assets supporting production are detailed below:

BioMarin Pharmaceutical Inc. (BMRN) - Canvas Business Model: Value Propositions

You're looking at the core value BioMarin Pharmaceutical Inc. (BMRN) delivers to patients and the market, which is anchored in first-in-class or best-in-class treatments for very rare genetic diseases. The numbers show a clear focus on maximizing the value of their established portfolio while managing newer assets.

Voxzogo (vosoritide) for achondroplasia, the only approved treatment

The primary value here is offering the only approved treatment for achondroplasia, a condition with significant unmet medical need due to respiratory, orthopedic, and mental health complications. This exclusivity drives strong commercial performance. For the full year 2025, BioMarin Pharmaceutical Inc. reaffirmed its outlook for VOXZOGO revenue to be between $900 million and $935 million. In the third quarter of 2025 alone, VOXZOGO brought in $218 million in sales, representing a 15% year-over-year growth. Year-to-date, VOXZOGO revenue increased by 24% compared to the same period in 2024. The global reach is expanding; as of the end of Q3 2025, children in 55 countries were being treated, tracking toward a goal of over 60 countries by 2027. Initiatives in the U.S. during Q3 2025 saw new patient starts across all ages, with the majority being children under 2 years of age.

Durable, life-saving enzyme replacement therapies for ultra-rare conditions

BioMarin Pharmaceutical Inc.'s foundation is built on these durable therapies for ultra-rare conditions, where high barriers to entry support strong pricing power. The Enzyme Therapies franchise, which includes PALYNZIQ, VIMIZIM, NAGLAZYME, BRINEURA, and ALDURAZME, is a significant value driver. Year-to-date in 2025, Total Enzyme Therapies revenue grew 8% year-over-year. PALYNZIQ, for phenylketonuria (PKU), marked its third consecutive quarter of 20%+ year-over-year growth, which the company attributes to greater numbers of patients titrating to their daily maintenance dose and strong adherence. For instance, in Q2 2025, PALYNZIQ revenue increased 20% year-over-year. To give you a sense of the scale and maturity of these assets, the MPS VI drug, Naglazyme, is still growing and is thought to have a peak sales potential approaching $530 million. The overall Enzyme Therapies business is a $2 billion-plus franchise.

Here's a quick look at the performance of some key enzyme therapies based on Q2 2025 data:

Single-administration gene therapy option (Roctavian) for severe hemophilia A

The value proposition for severe hemophilia A patients is a single-administration treatment, Roctavian (valoctocogene roxaparvovec-rvox), which offers the potential for years of bleed control. However, the commercial uptake has been slow due to reimbursement hurdles. In Q2 2025, Roctavian generated $9 million in sales, up from $7 million the prior year, with focus restricted to the U.S., Germany, and Italy. To create a path to profitability for this asset, BioMarin Pharmaceutical Inc. is actively pursuing options to divest ROCTAVIAN and remove it from the portfolio, having already reduced direct annual expenses to approximately $60 million starting in 2025. This strategic shift allows the company to redirect resources to its core growth drivers.

High-touch patient support programs for complex, chronic genetic disorders

For these complex, chronic genetic disorders, the value extends beyond the molecule itself to ensuring patients can successfully initiate and remain on therapy. This is critical for therapies like PALYNZIQ, where adherence and titration are key to efficacy. BioMarin Pharmaceutical Inc. supports this through dedicated programs, which you can see reflected in the reported adherence metrics.

• Strong adherence drives PALYNZIQ revenue growth, showing patient commitment.
• New patient starts for VOXZOGO in Q3 2025 included the youngest patients (under 2 years).
• Support is necessary for complex dosing titration schedules, like PALYNZIQ.
• The company generated operating cash flows of $369 million in Q3 2025, which helps fund these support structures.

The overall financial strength, with total cash and investments at approximately $2.0 billion at the end of Q3 2025, underpins the ability to maintain these high-touch services, even while executing a major acquisition of Inozyme for $270 million in March 2025.

BioMarin Pharmaceutical Inc. (BMRN) - Canvas Business Model: Customer Relationships

You're managing relationships in the ultra-rare disease space, which means every interaction has to be precise and deeply personal. BioMarin Pharmaceutical Inc. structures its customer relationships around this high-stakes, specialized environment.

High-touch, direct engagement with specialized treatment centers

BioMarin Pharmaceutical Inc. focuses its commercial efforts on a limited, specialized network of treatment centers capable of handling complex genetic therapies. This direct engagement model is necessary because the patient populations are small and geographically dispersed.

The company is actively expanding the reach of its flagship skeletal condition therapy, VOXZOGO, which is currently treating patients in over 55 countries as of late 2025. Management has a stated goal to increase this footprint to over 60 countries by the end of 2027. This global rollout requires intense, direct coordination with the specific centers that manage conditions like achondroplasia.

Here's a snapshot of the commercial focus driving this relationship intensity:

• VOXZOGO 2025 revenue guidance is projected between $900 million and $935 million.
• Q2 2025 Total Revenues reached $825 million, showing strong demand driving the relationship network.
• The company is progressing BMN 401 for ENPP1 Deficiency, a condition with an estimated global patient population of approximately 1,000-2,000.

Dedicated patient support programs for reimbursement and therapy access

For rare disease patients, the hurdle isn't just the science; it's navigating the complex payer landscape. BioMarin Pharmaceutical Inc. deploys its BioMarin RareConnections™ program specifically to address this. This service is designed to help patients and caregivers understand their insurance coverage and access financial assistance options.

This commitment extends beyond just treatment access into community support. For example, the RARE Scholars program, which supports U.S. college students living with specific rare genetic conditions, has awarded nearly $200,000 to 27 students since its start in 2018.

The support structure is comprehensive:

Long-term monitoring and follow-up for gene therapy patients

Gene therapies, like ROCTAVIAN for severe hemophilia A, demand a long-term relationship commitment due to their one-time treatment nature and the need to track durability and safety over many years. BioMarin Pharmaceutical Inc. has compelling data supporting this long-term engagement.

Five-year Phase 3 results from the GENEr8-1 trial for ROCTAVIAN showed sustained efficacy. After five years, 81.3% of participants remained off prophylaxis. Furthermore, the mean annualized bleeding rate was just 0.6 bleeds/year.

Key long-term durability metrics include:

• 77.8% of participants experienced zero treated bleeds during year five.
• Mean Factor VIII activity remained in the mild hemophilia range (24.0 IU/dL one-stage assay).
• The safety profile confirmed no new signals, inhibitors, or treatment-related malignancies across the five-year follow-up.

Medical Science Liaisons (MSLs) providing defintely specialized education

The MSLs at BioMarin Pharmaceutical Inc. act as peer scientific experts, focusing on in-depth, fair-balanced scientific exchange with key opinion leaders. Their role is critical for educating the specialized medical community on the science behind these complex treatments.

For instance, the Enzyme Conditions MSL team focuses on lysosomal storage disorders (LSDs) such as MPS IVA, MPS VI, and CLN2 disease. They are tasked with cultivating current and future thought leaders at local, regional, and national levels. This field-based team combines clinical expertise with market awareness to serve as a conduit of information for internal stakeholders, supporting business planning and development.

The MSL function is essential for niche areas where traditional marketing is restricted, such as in the EU for prescription drugs. They support the initiation and development of External Research proposals, which is a direct scientific relationship-building activity. Finance: draft 13-week cash view by Friday.

BioMarin Pharmaceutical Inc. (BMRN) - Canvas Business Model: Channels

You're looking at how BioMarin Pharmaceutical Inc. gets its specialized therapies to the right patients, which, for rare diseases, is never a simple over-the-counter transaction. The channel strategy is built around high-touch, expert engagement and complex logistics management.

Direct sales force targeting specialized pediatric endocrinologists and geneticists.

The core of getting complex treatments to market relies on a highly specialized, direct engagement model. BioMarin Pharmaceutical Inc. deploys a global force of approximately 500 representatives to connect directly with key opinion leaders and the specific treatment centers that handle rare genetic diseases. This direct channel is essential for explaining the intricate clinical data required for adoption. Furthermore, the company has secured a 98% coverage rate with major U.S. payers for its overall product portfolio, which is critical for channel success post-prescription.

Global network of specialty distributors and hospital pharmacies.

Moving these therapies requires more than just a prescription; it demands flawless logistics. BioMarin Pharmaceutical Inc. uses strategic partnerships with specialty distributors, naming leaders like McKesson and AmerisourceBergen in their channel strategy. This network manages the complex distribution and reimbursement hurdles inherent in specialty pharmaceuticals. To give you a sense of scale, the international segment of BioMarin Pharmaceutical Inc.'s business contributes over 45% of total product revenue, showing the importance of this global distribution reach.

Direct-to-patient services for complex, injectable therapies.

For therapies like their injectable products, BioMarin Pharmaceutical Inc. supports the patient journey directly through programs like BioMarin RareConnections™. This service coordinates with a specialty pharmacy to schedule medication delivery at a date and time convenient for the patient. The team also provides one-to-one financial navigation support to help families understand insurance coverage and identify financial assistance options they may qualify for. Historically, the RARE Scholars program has awarded nearly $200,000 to 27 students since 2018, showing a commitment to community support that underpins patient trust.

Focused commercial operations for Roctavian in the U.S., Germany, and Italy.

Following a strategic realignment announced in 2024, commercial efforts for the hemophilia A gene therapy, Roctavian, are strictly focused on three reimbursed markets: the U.S., Germany, and Italy. This focus is designed to streamline resource allocation. BioMarin Pharmaceutical Inc. anticipates reducing annual direct Roctavian expenses to approximately $60 million, beginning in 2025, with the goal of making the therapy profitable by the end of 2025. This contrasts with historical performance where the therapy generated only $7 million in sales under the broader strategy, though peak sales in these limited markets are modeled around $140 million.

Here's a quick look at how the channels support the overall financial picture as of late 2025:

The RareConnections™ program is designed to help patients navigate access, which is a crucial function for any specialty product channel. For instance, the coordination with specialty pharmacies for delivery is a key operational step following prescription authorization. You see the company's overall revenue growth, with Q2 2025 Total Revenues at $825 million, driven by strong demand across the portfolio, which validates the effectiveness of these targeted channels.

BioMarin Pharmaceutical Inc. (BMRN) - Canvas Business Model: Customer Segments

You're looking at the core patient populations BioMarin Pharmaceutical Inc. serves as of late 2025. This isn't about future potential; it's about the real-world patient base driving the current revenue, especially after the Q3 2025 update.

The customer segments are sharply defined by rare, genetically-driven conditions, which is where BioMarin Pharmaceutical Inc. focuses its commercial muscle. The financial data from the third quarter of 2025 clearly shows where the growth momentum is concentrated.

Pediatric and adult patients with achondroplasia (Voxzogo)

This segment is key to BioMarin Pharmaceutical Inc.'s near-term outlook. For the full year 2025, the company reaffirmed its revenue forecast for Voxzogo in the range of $900 million and $935 million. In the third quarter of 2025 alone, Voxzogo generated sales of $218 million, marking a 15% year-over-year increase. The worldwide incidence rate for achondroplasia is roughly one in every 25,000 live births. Critically, only about 25% of people with achondroplasia have growth plates still open, making them the target for Voxzogo treatment. As of the end of Q3 2025, children in 55 countries were on Voxzogo therapy. To be fair, about 75% of Voxzogo revenue comes from outside the U.S..

Patients with Phenylketonuria (PKU) requiring metabolic management (Palynziq)

This group is part of the larger Enzyme Therapies unit, which saw year-to-date growth driven by Palynziq. Palynziq injection sales in Q3 2025 hit $109 million, showing a strong 20% year-over-year jump. The condition itself, Phenylketonuria (PKU), or phenylalanine hydroxylase (PAH) deficiency, affects approximately 70,000 people in the regions where BioMarin Pharmaceutical Inc. operates. The company is also advancing a supplemental Biologics License Application for adolescents aged 12-17, indicating an expansion of this customer base is being pursued.

Patients with Mucopolysaccharidosis (MPS) disorders (e.g., MPS I, Morquio A)

This segment is served by products like Aldurazyme, Naglazyme, and Vimizim, all consolidated under Enzyme Therapies. While the overall Enzyme Therapies revenue grew 8% year-to-date in 2025, the Q3 2025 revenue for this franchise was up only 1% year-over-year, with Aldurazyme sales being lower compared to the prior year period. Naglazyme and Vimizim saw large orders in the second quarter, which made the sequential Q3 revenue look softer.

Here's a quick look at how the key revenue drivers performed in Q3 2025:

Patients with severe Hemophilia A (Roctavian, pending divestiture)

This segment is undergoing a strategic shift. BioMarin Pharmaceutical Inc. announced in October 2025 that it is pursuing options to divest Roctavian and remove it from its portfolio. The therapy generated $26 million in total sales in 2024 and only $23 million over the first nine months of 2025. Despite the divestiture pursuit, BioMarin Pharmaceutical Inc. will continue to make Roctavian commercially available in its three key markets-the U.S., Germany, and Italy-until the next steps are finalized. The company had previously hoped to cut its direct annual expenses for Roctavian to $60 million starting in 2025.

The focus for BioMarin Pharmaceutical Inc. is clearly shifting resources, as evidenced by the strategic pivot away from Roctavian and toward the growth assets. You can see the concentration of effort in the numbers:

• Voxzogo and Palynziq year-to-date 2025 revenue growth exceeded 20% each.
• Total Q3 2025 revenues were $776 million, up 4% year-over-year.
• The company generated operating cash flows of $369 million in the third quarter of 2025.

If onboarding takes 14+ days for these specialized treatments, patient adherence risk definitely rises.

Finance: draft 13-week cash view by Friday.

BioMarin Pharmaceutical Inc. (BMRN) - Canvas Business Model: Cost Structure

You're looking at the expense side of BioMarin Pharmaceutical Inc.'s operations as of late 2025. The costs here reflect a company balancing heavy investment in future growth with the execution of efficiency programs.

High R&D expenses to advance pipeline candidates like BMN 351 and BMN 333.

Research and Development spending remains a major cost driver, essential for advancing those novel therapies. For the twelve months ending September 30, 2025, BioMarin Pharmaceutical Inc.'s Research and Development Expenses hit $0.903B, marking a 15.78% jump year-over-year. This spend supports critical milestones, such as the expected initial proof-of-concept data for BMN 351, their Duchenne Muscular Dystrophy candidate, anticipated in the second half of 2025. To be fair, R&D spend can fluctuate; for instance, GAAP and Non-GAAP R&D expenses were lower year-over-year in the second quarter of 2025, but the third quarter saw a Y/Y increase driven by the $221 million In-Process Research & Development (IPR&D) charge related to the July 1, 2025, acquisition of Inozyme Pharma, Inc.

Significant cost of goods sold (COGS) due to complex biologic manufacturing.

Manufacturing complex biologics inherently carries a high Cost of Goods Sold (COGS). While we don't have the full 2025 COGS number yet, the impact of manufacturing and product mix is visible in the gross profit line. For example, in the third quarter of 2025, the company noted lower Cost of Sales due to a favorable product mix during that quarter. This suggests that managing the cost associated with producing therapies like VOXZOGO and the Enzyme Therapies portfolio is a constant focus area.

Selling, General, and Administrative (SG&A) costs for global commercial expansion.

Expanding commercial reach globally for products like VOXZOGO requires significant SG&A investment. In the second quarter of 2025, Non-GAAP SG&A actually increased year-over-year, which the company attributed to investment in its ERP implementation and business unit expansion initiatives. This trend continued into the third quarter of 2025, with Q3 GAAP and Non-GAAP SG&A expenses increasing year-over-year due to ongoing investment in business unit expansion. For context on the scale, advertising expenses alone for the full year 2024 were $34.5 million.

Here's a quick look at the latest reported expense figures we have for the nine months ending September 30, 2025, compared to the prior year:

Realizing benefits from a $500 million cost transformation program.

BioMarin Pharmaceutical Inc. is actively working to offset these high operating costs through structural changes. The company announced a $500 million cost transformation program back in September 2024. You should note that the full realization of benefits from this program is targeted for 2026, meaning the company is still in the implementation phase throughout 2025. This transformation, which includes enterprise-wide reorganization and external spend optimization, is designed to help the company achieve a 40% Non-GAAP Operating Margin in 2026.

The cost structure is clearly being managed for efficiency gains.

• Cost transformation implementation ongoing through 2025.
• Full cost benefit realization expected in 2026.
• Program aims to support a 40% Non-GAAP Operating Margin target for 2026.
• Organizational model is now built around three business units: Enzyme Therapies, Skeletal Conditions, and ROCTAVIAN.

Finance: draft 13-week cash view by Friday.

BioMarin Pharmaceutical Inc. (BMRN) - Canvas Business Model: Revenue Streams

You're looking at the core income drivers for BioMarin Pharmaceutical Inc. as we head into late 2025. The company has been laser-focused on its core portfolio, which is showing up clearly in the latest guidance figures.

The overall expectation for the top line is strong, reflecting the success of their key products. BioMarin Pharmaceutical Inc. has raised its Full-year 2025 Total Revenue Guidance to a range of $3.15 billion to $3.2 billion.

The primary engine driving this forecast is the continued global expansion of their achondroplasia treatment. They reaffirmed the Voxzogo product sales outlook, expecting it to land between $900 million to $935 million for the full year 2025. This product saw year-to-date revenue growth of 24% Y/Y as of the third quarter. It's defintely the star performer right now.

The foundation of the business remains the Sales of Enzyme Therapies, which management has characterized as a $2 billion-plus franchise over the last 12 months. This franchise includes established medicines like Palynziq, Vimizim, and Naglazyme. The momentum here is solid, with year-to-date growth contributing significantly to the overall results.

Here's a quick look at how the core revenue streams are performing based on recent updates:

You should also note the strategic move regarding the gene therapy asset. BioMarin Pharmaceutical Inc. announced the decision to pursue options to divest ROCTAVIAN and remove it from the portfolio as they focus on the core business units. This means the company is actively looking at out-licensing opportunities for this asset, which had limited commercial success. While the divestiture process is underway, the company is maintaining commercial availability in the U.S., Italy, and Germany.

Regarding other potential non-product revenue, the pursuit of the Roctavian divestiture is expected to involve a transition, but the search results do not specify an exact amount for any immediate milestone payments or licensing fees tied to this specific action as of late 2025. What this estimate hides is the impact of any one-time closing fees from the divestiture itself, which would likely be reported later. However, the company did record a significant, non-recurring charge:

• In-Process Research & Development (IPR&D) charge related to the Inozyme Pharma acquisition: $221 million on a pre-tax basis through Q3 2025.

The revenue focus is clearly on maximizing the growth trajectory of VOXZOGO and maintaining the high-margin performance of the established Enzyme Therapies portfolio.

Finance: draft 13-week cash view by Friday.