BIOMARIN PHARMACEUTICAL (BMRN) Company Report

BioMarin's CEO, Alexander Hardy, outlined a strategic reorganization focused on growth, innovation, and value commitment, including a $500 million cost transformation and a target of 40% non-GAAP operating margin in 2026, up from 19% in 2023.
The company anticipates several key pipeline readouts and regulatory decisions, including top-line results for BMN 351 for Duchenne muscular dystrophy by the end of 2025, Phase III data for Voxogo in hypochondroplasia and the 4.0.1 program at the beginning of 2026, and an FDA action date of February 28, 2026, for Palynziq in adolescents.
BioMarin expressed confidence in Voxogo's continued growth over the next several years, driven by existing achondroplasia markets and a potential hypochondroplasia launch in 2027, with an estimated addressable market of 14,000 patients globally for hypochondroplasia.
The company's business development strategy focuses on genetically defined conditions, leveraging its global footprint across 80 countries and specialized capabilities to acquire assets.

Nov 18, 2025, 10:30 AM

Transcript

BioMarin Provides Pipeline and Financial Strategy Update at Jefferies Conference

BMRN

Guidance Update

New Projects/Investments

Product Launch

BioMarin is targeting a 40% non-GAAP operating margin next year, a significant increase from 19% in 2023, driven by a $500 million cost transformation.
The company anticipates several key pipeline readouts and approvals, including BMN 351 for Duchenne muscular dystrophy top-line results by the end of this year, Voxogo for hypochondroplasia Phase 3 data early next year, and an FDA action date of February 28 of next year for Palynziq in adolescents.
BioMarin expects continued growth for Voxogo, with the hypochondroplasia indication representing an addressable market of 14,000 patients and a potential bolus launch in 2027.
The company has $2 billion in flexibility for business development, focusing on genetically defined conditions where its global footprint and capabilities provide a competitive advantage.
BioMarin is pursuing multiple avenues for intellectual property protection for Voxogo, including orphan drug exclusivity, International Trade Commission litigation, and potential traditional IP litigation in the U.S. and Europe.

Nov 18, 2025, 10:30 AM

Transcript

BioMarin's PALYNZIQ sBLA Accepted for Priority Review by FDA

BMRN

Product Launch

New Projects/Investments

The U.S. Food and Drug Administration (FDA) has accepted BioMarin Pharmaceutical Inc.'s supplemental Biologics License Application (sBLA) for PALYNZIQ (pegvaliase-pqpz) for Priority Review.
This sBLA seeks to expand treatment to adolescents aged 12-17 with phenylketonuria (PKU).
The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2026.
The application is based on positive results from the Phase 3 PEGASUS study, which demonstrated statistically significant blood phenylalanine (Phe) lowering compared to diet alone.
BioMarin also plans to share these data with the European Medicines Agency to expand treatment with PALYNZIQ to adolescents in the European Union.

Oct 29, 2025, 1:00 PM

Press Release

BioMarin Raises 2025 Revenue Guidance, Announces Roctavian Divestment, and Updates 2027 Outlook

BMRN

Guidance Update

M&A

Revenue Acceleration/Inflection

BioMarin raised its full-year 2025 total revenue guidance to $3.15 billion at the midpoint and reaffirmed its Voxzogo revenue outlook to be between $900 million and $935 million. The company also updated its full-year 2025 non-GAAP operating margin guidance to between 26% and 27% and non-GAAP diluted EPS guidance to between $3.50 and $3.60.
The company reported a year-to-date total revenue increase of 11% compared to 2024, driven by strong performance in its global enzyme therapies and skeletal conditions business units, and ended Q3 2025 with approximately $2 billion in cash and investments.
BioMarin announced the decision to pursue options to divest Roctavian to focus on business units aligned with its strategic priorities.
The previously provided 2027 revenue outlook was updated, with the company no longer providing a specific estimate due to market unknowns and potential Voxzogo competition; however, the lower end of its range of estimates is in line with current 2027 top-line consensus (excluding Roctavian), and higher-end scenarios could reach $4 billion (excluding Roctavian).

Oct 27, 2025, 8:30 PM

Transcript

BioMarin Updates 2025 Guidance, Announces ROCTAVIAN Divestment

BMRN

Earnings

Guidance Update

M&A

BioMarin raised its full-year 2025 total revenue guidance at the midpoint and reaffirmed its VOXOGO revenue outlook for 2025, which is expected to be between $900 million and $935 million.
The company reported a cash and investments balance of approximately $2 billion at the end of Q3 2025.
BioMarin announced its decision to pursue options to divest ROCTAVIAN to focus on business units aligned with its strategic priorities.
Full-year 2025 non-GAAP operating margin guidance was updated to between 26-27% and non-GAAP diluted earnings per share guidance to between $3.5 and $3.6.
Q3 2025 results included a $221 million charge for acquired in-process research and development (IPR&D), which impacted diluted earnings per share by approximately $1.1 per share.

Oct 27, 2025, 8:30 PM

Transcript

BioMarin Reports Strong Q3 2025 Results, Updates Full-Year Guidance, and Plans Roctavian Divestment

BMRN

Earnings

Guidance Update

New Projects/Investments

BioMarin reported strong Q3 2025 results, with year-to-date total revenue up 11% compared to 2024, and a cash and investments balance of approximately $2 billion at the end of the third quarter.
The company raised the lower end of its full-year 2025 total revenue guidance to $3.15 billion and reaffirmed its Voxzogo revenue outlook of $900 million to $935 million for 2025.
Full-year 2025 non-GAAP operating margin guidance was updated to 26% to 27%, and non-GAAP diluted EPS guidance to $3.50 to $3.60, reflecting a $221 million Q3 IPR&D charge related to an acquisition.
BioMarin announced its decision to pursue options to divest Roctavian to focus on business units aligned with strategic priorities.
The previous 2027 revenue outlook was rescinded, with a new range indicating the lower end is in line with current consensus and the higher end could reach $4 billion, both excluding Roctavian, due to competitive uncertainties.

Oct 27, 2025, 8:30 PM

Transcript

BioMarin Updates 2025 Guidance, Announces ROCTAVIAN Divestment, and Revises 2027 Revenue Outlook

BMRN

Guidance Update

M&A

Revenue Acceleration/Inflection

BioMarin raised its full-year 2025 total revenue guidance to a lower end of $3.15 billion and updated non-GAAP diluted EPS guidance to between $3.50 and $3.60, and non-GAAP operating margin guidance to between 26% and 27%.
The company announced its decision to pursue options to divest ROCTAVIAN from its portfolio to focus on strategic priorities.
VOXZOGO revenue is projected to exceed $900 million in 2025, reflecting 25% growth at the midpoint of guidance, with year-to-date growth of 24% compared to 2024.
The previously provided 2027 revenue outlook was rescinded due to market uncertainties, with the company indicating a range where the lower end aligns with current consensus and the higher end could reach $4 billion, both excluding ROCTAVIAN.
BioMarin ended Q3 2025 with approximately $2 billion in cash and investments and generated $728 million in operating cash flow year-to-date.

Oct 27, 2025, 8:30 PM

Transcript

BioMarin Pharmaceutical Inc. Reports Q3 2025 Results, Raises Revenue Guidance

BMRN

Earnings

Guidance Update

New Projects/Investments

BioMarin reported Q3 2025 year-to-date Total Revenues of $2,347 million, an 11% increase year-over-year, and raised its full-year 2025 Total Revenues guidance to between $3,150 million and $3,200 million.
The company updated its full-year 2025 Non-GAAP Operating Margin guidance to 26% - 27% and Non-GAAP Diluted EPS guidance to $3.50 - $3.60, primarily due to a $221 million impact from acquired IPR&D charges.
VOXZOGO revenue grew 24% year-to-date and its full-year 2025 revenue outlook was reaffirmed at $900 million to $935 million. Enzyme Therapies also saw 8% year-to-date revenue growth.
BioMarin plans to divest ROCTAVIAN and is advancing its pipeline, with a Phase 2/3 study for BMN 333 (targeting a superior achondroplasia treatment) expected to begin in 1H 2026, and VOXZOGO for hypochondroplasia Phase 3 data expected in 1H 2026.

Oct 27, 2025, 8:30 PM

Presentation

BioMarin Reports Q3 2025 Results, Raises Revenue Guidance, and Plans ROCTAVIAN Divestiture

BMRN

Earnings

Guidance Update

M&A

BioMarin Pharmaceutical Inc. reported total revenues of $776 million for Q3 2025, an increase of 4% compared to the same period in 2024, with year-to-date total revenues increasing 11%.
The company recorded a GAAP Net Loss of $(31) million for Q3 2025, primarily due to a $221 million In-Process Research & Development (IPR&D) charge associated with the acquisition of Inozyme Pharma, Inc..
BioMarin raised its full-year 2025 Total Revenues guidance at the midpoint to a range of $3,150 million to $3,200 million. However, it updated its Non-GAAP Operating Margin % to 26% to 27% and Non-GAAP Diluted EPS to $3.50 to $3.60, reflecting the impact of the IPR&D charge.
The company announced its decision to pursue options to divest ROCTAVIAN from its portfolio.

Oct 27, 2025, 8:08 PM

8-K

BioMarin Reports Q3 2025 Results, Updates Full-Year Guidance, and Plans ROCTAVIAN Divestiture

BMRN

Earnings

Guidance Update

M&A

BioMarin reported total revenues of $776 million for the third quarter of 2025, representing a 4% increase compared to the same period in 2024, primarily driven by strong revenue growth in VOXZOGO and PALYNZIQ.
The company announced its decision to pursue options to divest ROCTAVIAN from its portfolio, evaluating out-licensing opportunities for the gene therapy.
For the third quarter of 2025, BioMarin recorded a GAAP Net Loss of $31 million and Non-GAAP Income of $22 million, largely impacted by a $221 million In-Process Research & Development (IPR&D) charge related to the acquisition of Inozyme Pharma, Inc..
BioMarin raised its full-year 2025 total revenues guidance at the midpoint to a range of $3,150 million to $3,200 million and updated its Non-GAAP Diluted EPS guidance to $3.50 to $3.60.

Oct 27, 2025, 8:03 PM

Press Release

Earnings Transcripts

Quarterly earnings call transcripts for BIOMARIN PHARMACEUTICAL.

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