Core Insights - BioMarin Pharmaceutical reported $776.13 million in revenue for Q3 2025, a 4.1% year-over-year increase, but fell short of the Zacks Consensus Estimate of $784.4 million, resulting in a surprise of -1.05% [1] - The company’s EPS for the quarter was $0.12, significantly lower than the $0.91 reported a year ago, but exceeded the consensus estimate of -$0.15, resulting in an EPS surprise of +180% [1] Revenue Breakdown - ALDURAZYME generated $54 million, surpassing the average estimate of $45.06 million from 11 analysts [4] - KUVAN reported $24 million, exceeding the average estimate of $21.1 million, but showed a year-over-year decline of -14.3% [4] - NAGLAZYME achieved $122 million, slightly below the estimated $123.14 million, reflecting a -7.6% change year-over-year [4] - VIMIZIM brought in $183 million, which was lower than the average estimate of $192.42 million, but represented a +2.8% year-over-year increase [4] - Royalty and other revenues totaled $15.32 million, exceeding the average estimate of $11.31 million, marking a +29% year-over-year increase [4] - PALYNZIQ generated $109 million, surpassing the average estimate of $103.1 million, with a year-over-year increase of +19.8% [4] - VOXZOGO reported $218 million, slightly below the average estimate of $233.96 million, but showed a +14.7% year-over-year increase [4] - ROCTAVIAN's revenue was $3 million, significantly lower than the estimated $9.39 million, reflecting a -57.1% year-over-year decline [4] - Total net product revenues were $760.81 million, below the average estimate of $778.49 million, but represented a +3.7% year-over-year increase [4] - BRINEURA achieved $48 million, exceeding the average estimate of $43.28 million, with a year-over-year increase of +29.7% [4] Stock Performance - BioMarin's shares returned +1.2% over the past month, underperforming the Zacks S&P 500 composite's +2.5% change [3] - The stock currently holds a Zacks Rank 4 (Sell), indicating potential underperformance relative to the broader market in the near term [3]

Core Viewpoint - BioMarin Pharmaceutical plans to divest its gene therapy product, which was once anticipated to be a blockbuster treatment for a rare bleeding disorder [1] Company Summary - The gene therapy in question was previously expected to generate significant revenue as a leading treatment option for a specific rare bleeding disorder [1]

Core Insights - BioMarin Pharmaceutical reported quarterly earnings of $0.12 per share, exceeding the Zacks Consensus Estimate of a loss of $0.15 per share, but down from $0.91 per share a year ago [1] - The earnings surprise was +180.00%, and the company has surpassed consensus EPS estimates in all four of the last quarters [2] - Revenue for the quarter was $776.13 million, slightly missing the Zacks Consensus Estimate by 1.05%, but up from $745.74 million year-over-year [3] Earnings Performance - The company has consistently exceeded consensus EPS estimates, achieving this in four consecutive quarters [2] - The current consensus EPS estimate for the upcoming quarter is $0.97, with expected revenues of $819.69 million, and for the current fiscal year, the EPS estimate is $3.54 on revenues of $3.18 billion [8] Stock Performance and Outlook - BioMarin shares have declined approximately 17.1% year-to-date, contrasting with the S&P 500's gain of 15.5% [4] - The stock currently holds a Zacks Rank 4 (Sell), indicating expectations of underperformance in the near future [7] Industry Context - The Medical - Biomedical and Genetics industry, to which BioMarin belongs, is currently ranked in the top 38% of over 250 Zacks industries, suggesting a favorable industry outlook [9] - The performance of BioMarin's stock may be influenced by the overall industry outlook and trends in earnings estimate revisions [6][9]

Financial Data and Key Metrics Changes - BioMarin reported an 11% year-over-year increase in total revenues, leading to an updated full-year 2025 total revenue guidance of $3.15 billion, reflecting double-digit growth [5][11][14] - The company’s cash and investments balance reached approximately $2 billion at the end of Q3 2025, with operating cash flow of $369 million for the quarter and $728 million year-to-date [5][14] - Non-GAAP diluted earnings per share guidance was updated to between $3.50 and $3.60, with non-GAAP operating margin guidance revised to between 26% and 27% [14][15] Business Line Data and Key Metrics Changes - The enzyme therapies business unit achieved 8% year-to-date growth, with PALYNZIQ driving over 20% growth [11][17] - VOXZOGO, the treatment for achondroplasia, is expected to generate over $900 million in revenue for 2025, representing a 25% growth at the midpoint of guidance [7][11] - VOXZOGO revenue increased by 24% year-to-date compared to 2024, with a 15% growth in Q3 alone [11][18] Market Data and Key Metrics Changes - Approximately 75% of year-to-date VOXZOGO revenue was generated outside the U.S., indicating strong international demand [19][20] - The company is expanding its global footprint, with VOXZOGO now available in 55 countries, and anticipates significant growth opportunities as it penetrates new markets [18][19] Company Strategy and Development Direction - BioMarin is focusing on strategic investments in enzyme therapies and skeletal conditions, while divesting from non-core assets like ROCTAVIAN [9][10] - The company is preparing for the launch of VOXZOGO's second indication for hypochondroplasia, with pivotal data expected in the first half of 2026 [8][21] - BioMarin aims to enhance its business development strategy by pursuing early-stage collaborations and phase III assets to drive growth [43][44] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving record commercial results for the full year, despite competitive pressures and market dynamics [5][10] - The company acknowledged the uncertainty surrounding potential competition for VOXZOGO, leading to a range of revenue scenarios for 2027 [15][33] - Management emphasized the importance of maintaining strong patient adherence and expanding the prescriber base to sustain growth [20][69] Other Important Information - BioMarin announced a $221 million charge for acquired in-process research and development related to the Enzyme Pharma acquisition, impacting both GAAP and non-GAAP financials [12][13] - The company is advancing its pipeline with multiple data readouts and regulatory milestones expected in the coming quarters [30][31] Q&A Session Summary Question: Can you elaborate on the 2027 guidance and the changes since it was initially issued? - Management indicated that the original $4 billion 2027 outlook was reassessed due to potential VOXZOGO competition and other market dynamics, leading to a range of estimates rather than a specific forecast [33][34][35] Question: Why were VOXZOGO sales down quarter-over-quarter? - Management attributed the decline to timing shifts in larger orders and reaffirmed the total VOXZOGO revenue range for the year, emphasizing steady patient additions across all markets [39][41][42] Question: What is the strategy for business development given the focus on VOXZOGO? - Management highlighted the importance of business development as a priority, with ongoing efforts to pursue deals that could enhance the company's portfolio [43][44] Question: What are the expectations for the upcoming BMN-333 data? - Management indicated that they are looking for significant increases in exposure levels compared to VOXZOGO to demonstrate clinical superiority [53][54] Question: Can you provide insights on the long-term growth targets and cash flow expectations? - Management confirmed that the target for 40% non-GAAP operating margins remains, but acknowledged that it is dependent on top-line performance [59][60]

Financial Data and Key Metrics Changes - BioMarin reported an 11% year-over-year increase in total revenue, leading to an updated full-year revenue guidance of $3.15 billion, reflecting double-digit growth [5][11][15] - The company’s cash and investments balance reached approximately $2 billion at the end of Q3 2025, with operating cash flow of $369 million for the quarter and $728 million year-to-date [5][15] - Non-GAAP diluted earnings per share guidance was updated to between $3.50 and $3.60, with non-GAAP operating margin guidance adjusted to between 26% and 27% [15][16] Business Line Data and Key Metrics Changes - The enzyme therapies business unit achieved 8% year-to-date growth, primarily driven by Palynziq, although Q3 revenue was flat compared to Q3 2024 due to prior large orders [11][12] - Voxzogo, the treatment for achondroplasia, is expected to generate over $900 million in revenue for 2025, reflecting a 25% growth at the midpoint of guidance, with a year-to-date increase of 24% [7][11][19] - Palynziq also saw over 20% growth year-to-date, indicating strong demand and adherence among patients [11][18] Market Data and Key Metrics Changes - Approximately 75% of year-to-date Voxzogo revenue was generated outside the U.S., highlighting significant international growth opportunities [21][22] - The company is expanding its global footprint, with Voxzogo now available in 55 countries, and anticipates Q4 to be the highest revenue quarter for Voxzogo due to large contracted orders [19][21] Company Strategy and Development Direction - BioMarin is focusing on strategic investments in enzyme therapies and skeletal conditions, while also pursuing options to divest Roctavian to concentrate on core business units [9][10] - The company is preparing for the launch of Voxzogo's second indication for hypochondroplasia, with pivotal data expected in the first half of 2026 [8][23] - BioMarin aims to enhance its leadership in skeletal conditions by pursuing multiple indications for Voxzogo and advancing its next-generation product, BMN-333 [23][31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving record commercial results for the full year, despite challenges from potential competition for Voxzogo [5][10] - The company acknowledged the uncertainty surrounding 2027 revenue projections due to competitive dynamics but remains committed to executing its growth strategy [17][39] - Management emphasized the importance of business development as a priority, with ongoing efforts to identify and acquire under-resourced assets [46][52] Other Important Information - BioMarin announced a $221 million charge for acquired in-process research and development related to the Enzyme Pharma acquisition, impacting both GAAP and non-GAAP financials [12][14] - The company is focused on improving early diagnosis for hypochondroplasia and expanding treatment access through various initiatives [25][26] Q&A Session Summary Question: Can you elaborate on the 2027 guidance and the changes since it was issued? - Management indicated that the original $4 billion 2027 outlook was reassessed due to potential competition for Voxzogo and other market dynamics, leading to a range of scenarios rather than a specific forecast [36][38] Question: Why were Voxzogo sales down quarter-over-quarter? - Management attributed the decline to timing shifts in larger orders and reaffirmed the overall revenue guidance for Voxzogo, emphasizing steady patient additions across all markets [42][44] Question: What is the strategy for business development moving forward? - Management highlighted a strong focus on early-stage collaborations and the pursuit of phase three and commercial assets, aiming to leverage BioMarin's capabilities to enhance value [46][52] Question: What are the expectations for the upcoming BMN-333 data? - Management indicated that they are looking for significant pharmacokinetic improvements over Voxzogo, with a focus on achieving clinical superiority in future trials [56][57]

Financial Data and Key Metrics Changes - BioMarin reported an 11% year-over-year increase in total revenue, with cash and investments reaching approximately $2 billion at the end of Q3 2025 [5][6][15] - The company raised its full-year 2025 total revenue guidance to a range of $3.15 billion, reflecting strong performance and operational efficiencies [12][15] - Non-GAAP diluted earnings per share guidance was updated to between $3.50 and $3.60, with an operating margin guidance of 26% to 27% [15][17] Business Line Data and Key Metrics Changes - The Enzyme Therapies business unit experienced 8% year-to-date growth, with Palynziq and VoxoGo each increasing by over 20% year-to-date [12][19] - VoxoGo revenue is expected to reach between $900 million and $935 million for the full year 2025, with a 24% increase year-to-date compared to 2024 [6][12][20] - The Skeletal Conditions business unit, driven by FOXOGO, is projected to generate over $900 million in revenue for 2025, representing a 25% growth at the midpoint of guidance [6][19] Market Data and Key Metrics Changes - Approximately 75% of year-to-date VoxoGo revenue was generated outside the U.S., indicating strong international demand [20][21] - VoxoGo is now available in 55 countries, with significant growth in patient numbers quarter-over-quarter [20][21] Company Strategy and Development Direction - BioMarin is focusing on strategic investments in the Enzyme Therapies and Skeletal Conditions business units while pursuing options to divest ROCTAVIAN [9][10] - The company aims to expand its global footprint and deepen penetration in existing markets, particularly for VoxoGo and upcoming indications [20][21] - Future growth is anticipated through new product launches and business development opportunities, with a focus on advancing internal pipelines [11][15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving record commercial results for the full year, despite challenges from potential competition [5][11] - The company is monitoring various market dynamics and has developed multiple scenarios for future revenue, particularly concerning VoxoGo competition [17][42] - Management emphasized the importance of maintaining value-creating activities while pursuing operational efficiencies [75] Other Important Information - A charge of $221 million for acquired in-process research and development related to the Enizyme Pharma acquisition impacted Q3 results [14] - The company is preparing for pivotal data readouts and potential new approvals in the coming quarters, particularly for hypochondroplasia and BMN 333 [36][37] Q&A Session Summary Question: Can you elaborate on the 2027 guidance and the changes since it was initially issued? - Management indicated that various factors, including potential VoxoGo competition and the acquisition of Inozyme, influenced the revised outlook, leading to a range of estimates rather than a specific forecast [39][42] Question: Why were VoxoGo sales down quarter-over-quarter? - Management noted that the decline was due to timing shifts in larger orders and reaffirmed the total revenue range for VoxoGo for the year [48][50] Question: What is the company's strategy for business development? - Management highlighted a focus on early-stage collaborations and Phase III assets, emphasizing that business development remains a high priority [52][54] Question: What are the expectations for the upcoming BMN 333 data? - Management stated that they are looking for significant increases in free CNP levels to demonstrate clinical superiority over VoxoGo [64][66] Question: Can you clarify the 2027 cash flow targets? - Management confirmed that the cash flow targets are tied to revenue scenarios and emphasized ongoing optimization initiatives [79][80]

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Financial Performance - Year-to-date 2025 total revenues increased by 11% year-over-year, driven by over 20% year-over-year revenue growth for PALYNZIQ and VOXZOGO[11] - The company raised full-year 2025 total revenues guidance to between $3150 million and $3200 million, representing 11% fiscal year 2025 growth year-over-year[13] - VOXZOGO's fiscal year 2025 revenue outlook is reaffirmed to be between $900 million and $935 million[13] - Q3 2025 GAAP operating margin was negative 60%, a decrease of 213 percentage points year-over-year, while non-GAAP operating margin was 28%, a decrease of 249 percentage points year-over-year[21] - Q3 2025 GAAP diluted EPS was negative $016, a decrease of 129%, while non-GAAP diluted EPS was $012, a decrease of 87%[21] - Operating cash flow for Q3 2025 was $369 million, an increase of 66%[21] - Full-year 2025 non-GAAP operating margin guidance updated to 26% - 27% and non-GAAP diluted EPS guidance updated to $350 - $360[24] Product Performance - VOXZOGO Q3 year-to-date revenue increased 24% year-over-year[20] - Enzyme Therapies Q3 year-to-date revenue increased 8% year-over-year[20] - VOXZOGO Q3 2025 revenue was $218 million, a 15% increase year-over-year, and Q3 2025 year-to-date revenue was $654 million, a 24% increase year-over-year[33] Research and Development - The company is preparing for VOXZOGO in hypochondroplasia (HCH) launch, with pivotal data readout expected in the first half of 2026[38]

Exhibit 99.1 Contact: Investors: Media: Traci McCarty Marni Kottle BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc. (415) 455-7558 (650) 374-2803 BioMarin Reports Third Quarter 2025 Results and Provides Corporate Update Raises Full-year 2025 Total Revenues Guidance at the Midpoint; Reaffirms VOXZOGO Full-year Outlook 2025 Year-to-date Total Revenues Increased 11% Y/Y Led by More Than 20% Revenue Growth for PALYNZIQ and VOXZOGO BioMarin Focuses Commercial Portfolio; Pursuing Options to Divest ROCTAV ...

Core Insights - BioMarin Pharmaceutical Inc. raised its full-year 2025 total revenues guidance at the midpoint, reflecting strong demand for its therapies, particularly VOXZOGO and PALYNZIQ, which saw over 20% revenue growth [1][6][11] - The company reported a GAAP net loss of $31 million for Q3 2025, a significant decline from a net income of $106 million in Q3 2024, primarily due to a $221 million in-process research and development charge related to the acquisition of Inozyme Pharma [6][7][11] - BioMarin plans to divest ROCTAVIAN while continuing to evaluate out-licensing options for the gene therapy, aligning with its strategic focus on core business units [2][6][11] Financial Performance - Total revenues for Q3 2025 were $776 million, a 4% increase from $746 million in Q3 2024, driven by strong growth in VOXZOGO and PALYNZIQ [6][7] - Year-to-date total revenues increased by 11% to $2.347 billion compared to $2.107 billion in the same period last year [7][11] - Non-GAAP income for Q3 2025 decreased to $22 million from $178 million in Q3 2024, reflecting the impact of the IPR&D charge [6][7] Product Performance - VOXZOGO revenue for Q3 2025 was $218 million, up 15% year-over-year, with year-to-date revenue increasing 24% to $654 million [7][11] - PALYNZIQ achieved a 20% year-over-year growth in Q3 2025, with total enzyme therapies revenue growing 8% year-to-date [6][7] - The company is advancing its pipeline with several upcoming pivotal data readouts and potential product launches, including BMN 333 and BMN 401 [6][11] Strategic Focus - BioMarin is concentrating on its Enzyme Therapies and Skeletal Conditions business units, which are central to its growth strategy [2][6] - The company aims to expand VOXZOGO access in over 60 countries by 2027, with significant uptake in markets outside the U.S. [6][11] - The decision to divest ROCTAVIAN is part of a broader strategy to streamline its portfolio and focus on high-potential therapies [2][6]