BMRN Q3 Deep Dive: Product Expansion and Portfolio Shifts Define Outlook

Biotech company BioMarin Pharmaceutical (NASDAQ:BMRN) met Wall Street’s revenue expectations in Q3 CY2025, with sales up 4.1% year on year to $776.1 million. The company’s outlook for the full year was close to analysts’ estimates with revenue guided to $3.18 billion at the midpoint. Its non-GAAP profit of $0.12 per share was 62.5% below analysts’ consensus estimates.

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StockStory’s Take

BioMarin Pharmaceutical’s third quarter results reflected steady top-line growth, with management highlighting enzyme therapies and the Skeletal Conditions business as main contributors. CEO Alexander Hardy credited global demand for therapies like VOXZOGO and PALYNZIQ, noting, “These strong results are driven by the performance of our global enzyme therapies and Skeletal Conditions business units as we deliver for patients around the world.” The company’s operating margins declined due to higher R&D and SG&A expenses, partly from the Inozyme Pharma acquisition. Management acknowledged that this charge, along with business unit investments, weighed on profitability.

Looking forward, BioMarin’s updated guidance focuses on sustained growth from established products and expansion into new indications. Management emphasized upcoming milestones for VOXZOGO in hypochondroplasia and the advancement of next-generation therapies such as BMN 333. CFO Brian Mueller noted that future scenarios depend on competitive dynamics and pipeline execution, stating, “There are a number of outcomes, highly uncertain at this time, and therefore narrowing the range or coming up with more specific point-in-time estimates isn’t appropriate.” Expansion into new markets and continued business development remain strategic priorities.

Key Insights from Management’s Remarks

Management attributed Q3 performance to robust demand for key therapies, strategic business realignment, and ongoing investments in product development and commercialization.

• VOXZOGO drives growth: The Skeletal Conditions business, led by VOXZOGO for achondroplasia, saw year-to-date revenue up 24%, with management reaffirming its status as the standard of care and highlighting global expansion into 55 countries.
• Enzyme therapies steady: The Enzyme Therapies business unit maintained strong patient adherence and continued to add new patient starts, with PALYNZIQ showing more than 20% year-to-date growth and label expansion efforts underway for adolescent use.
• ROCTAVIAN divestiture underway: BioMarin announced plans to divest its gene therapy ROCTAVIAN, focusing resources on core growth areas and aiming to ensure continued access for patients in the U.S., Italy, and Germany during the transition period.
• Inozyme Pharma acquisition impact: The recent acquisition resulted in a significant in-process R&D charge, increasing both R&D and SG&A spending, but also added new pipeline assets like BMN 401 for ENPP1 deficiency, with pivotal data expected in 2026.
• Portfolio streamlining: Management discontinued several research programs that did not meet advancement criteria, aligning the business around high-potential assets and driving operational efficiency for future growth.

Drivers of Future Performance

BioMarin’s outlook centers on expanding its skeletal conditions franchise, navigating competitive pressures, and leveraging new business development opportunities to drive long-term growth.

• Pipeline advancement critical: Management is banking on successful late-stage clinical trials for VOXZOGO in hypochondroplasia and BMN 333, which could extend the company’s leadership in skeletal disorders and create new revenue streams.
• Competitive landscape evolving: The company expects increased competition in achondroplasia and related indications, with future scenarios modeled on different competitor launch timelines and market share impacts, potentially affecting revenue growth rates.
• Business development as a lever: Strategic acquisitions and partnerships remain top priorities, with available capital earmarked for assets that can be scaled by BioMarin’s global infrastructure, rather than for share buybacks or unrelated investments.

Catalysts in Upcoming Quarters

Looking ahead, StockStory’s team will closely monitor (1) data readouts for VOXZOGO in hypochondroplasia and progress on the BMN 333 program, (2) the execution and commercial impact of the Inozyme acquisition, and (3) BioMarin’s ability to advance business development initiatives. Pipeline milestones and competitive developments will also be key to tracking future performance.

BioMarin Pharmaceutical currently trades at $53.25, up from $52.70 just before the earnings. Is the company at an inflection point that warrants a buy or sell? The answer lies in our full research report (it’s free for active Edge members).

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