BioMarin’s first quarter performance was shaped by robust global demand for its rare disease therapies, particularly VOXZOGO, which continued its double-digit growth trajectory. Management attributed the healthy top-line results to strong patient uptake across 49 countries and the expanding footprint of its enzyme therapies, including notable gains for PALYNZIQ and ALDURAZYME. CEO Alexander Hardy explained, "These results pave the way for record full year performance in 2025 and boost cash generation for reinvestment in innovation and expansion." The company also highlighted operational discipline and the early benefits of last year’s strategic transformation, which contributed to significant profitability expansion.

Is now the time to buy BMRN? Find out in our full research report (it’s free).

While we enjoy listening to the management's commentary, our favorite part of earnings calls are the analyst questions. Those are unscripted and can often highlight topics that management teams would rather avoid or topics where the answer is complicated. Here is what has caught our attention.

Our Top 5 Analyst Questions BioMarin Pharmaceutical’s Q1 Earnings Call

• Philip Nadeau (TD Cowen) questioned if VOXZOGO’s Q2 revenue might decline and how future tariffs could affect financials. CFO Brian Mueller clarified that order timing, not price or demand, drives quarterly revenue variation, and that existing tariff exposure is minimal.
• Tommie Reerink (Goldman Sachs) asked about business development opportunities and the timing for BMN 333 data. CEO Alexander Hardy confirmed a focus on clinical-stage assets aligned with core strengths and expects a business development deal this year, with BMN 333 data updates planned for later in 2025.
• Jenny Gonzalez-Armenta (Leerink Partners) probed strategies for increasing VOXZOGO market penetration. Chief Commercial Officer Cristin Hubbard outlined initiatives to expand prescriber outreach, particularly in pediatric endocrinology, and efforts to increase patient access across age groups.
• Zaki Molvi (Jefferies) inquired about safety concerns for BMN 351 in Duchenne Muscular Dystrophy. Chief R&D Officer Greg Friberg acknowledged class-related toxicity risks and emphasized ongoing safety monitoring, with detailed data expected in the second half of the year.
• Jessica Fye (JPMorgan) raised concerns about VOXZOGO’s long-term value amid potential competition. Hubbard and Friberg responded by highlighting the drug’s robust safety and efficacy data, global reach, and plans for lifecycle management, including next-generation therapies.

Catalysts in Upcoming Quarters

In the coming quarters, our analysts are watching (1) the pace of VOXZOGO adoption in new and existing markets, especially as commercial efforts intensify in the U.S.; (2) regulatory progress and clinical data releases for key pipeline assets, including PALYNZIQ and BMN 333; and (3) the impact of increased R&D and commercial investments on margin trends. Updates on business development activity and responses to evolving international trade policies will also be critical signals.

BioMarin Pharmaceutical currently trades at $55.42, down from $62.77 just before the earnings. In the wake of this quarter, is it a buy or sell? The answer lies in our full research report (it’s free).

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