Biotech company BioMarin Pharmaceutical (NASDAQ: BMRN) reported Q2 CY2025 results topping the market’s revenue expectations, with sales up 15.9% year on year to $825.4 million. The company’s full-year revenue guidance of $3.16 billion at the midpoint came in 0.6% above analysts’ estimates. Its non-GAAP profit of $1.44 per share was 42.3% above analysts’ consensus estimates.

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StockStory’s Take

BioMarin Pharmaceutical delivered a second quarter that surpassed Wall Street’s expectations, with management attributing the strong results to double-digit revenue growth across its portfolio and significant progress in both commercial execution and pipeline development. CEO Alexander Hardy cited robust demand for key therapies, notably the global expansion of VOXZOGO and strong performance in enzyme therapies such as PALYNZIQ and VIMIZIM. Management credited business unit structure changes and enhanced patient support initiatives for new patient starts and adherence, particularly in younger cohorts. Cristin Hubbard, Chief Commercial Officer, highlighted, “We doubled the number of leads generated year-to-date year-over-year, which translated to an increase in net new U.S. patients.”

Looking forward, BioMarin’s guidance for the remainder of 2025 is driven by expected continued growth in VOXZOGO, expansion into new indications, and the integration of newly acquired pipeline assets. Management emphasized that investments in research, development, and commercial initiatives—including upcoming Phase III data for hypochondroplasia and the advancement of BMN 333—are central to sustaining momentum. CFO Brian Mueller noted that increased business unit investments could lead to higher operating expenses in the back half of the year, but maintained confidence in the company’s ability to deliver on its updated targets, stating, “We are raising full year 2025 non-GAAP earnings per share guidance to between $4.40 and $4.55.”

Key Insights from Management’s Remarks

Management pointed to strong global demand, successful product expansion, and a completed acquisition as key contributors to the quarter’s performance and outlook.

• VOXZOGO uptake in young children: Management highlighted rapid adoption and adherence in the 0-4-year-old cohort in the U.S., driven by expanded field force and digital promotion. The company attributed a doubling in year-to-date lead generation to these efforts, translating to more new patient starts.

• Enzyme therapy portfolio growth: PALYNZIQ and VIMIZIM both delivered notable year-over-year growth, with PALYNZIQ experiencing continued titration to maintenance doses and VIMIZIM benefiting from global demand and large regional orders. Management expects these trends to persist, supported by BioMarin’s patient support programs and global reach.

• Inozyme acquisition completion: The acquisition of Inozyme broadened BioMarin’s enzyme therapy portfolio, adding BMN 401 for ENPP1 deficiency. Management emphasized the rapid integration and anticipated pivotal data readout in the first half of 2026 as a strategic milestone.

• Pipeline progress and upcoming milestones: BMN 333, a next-generation therapy for achondroplasia, achieved a target pharmacokinetic profile in early studies, prompting advancement to Phase II/III. Management also cited upcoming regulatory submissions for PALYNZIQ in adolescents and pivotal data for VOXZOGO in hypochondroplasia as near-term drivers.

• Strategic business development: Leadership reiterated ongoing efforts to enhance the pipeline through targeted acquisitions, with a focus on diversifying growth and supporting long-term revenue expansion. They signaled continued evaluation of opportunities to augment the portfolio beyond the Inozyme transaction.

Drivers of Future Performance

BioMarin’s outlook is supported by new product launches, label expansions, and continued investment in pipeline assets, balanced against rising expenses and evolving market dynamics.

• VOXZOGO indication expansion: Management expects the upcoming Phase III data in hypochondroplasia and continued efforts to improve early diagnosis to drive new patient growth. The company is actively engaging with the medical community to enhance disease awareness and support potential launches in additional indications.

• Pipeline asset advancement: The progress of BMN 333 into Phase II/III and BMN 401’s pivotal readout are expected to be central to BioMarin’s growth strategy. Management believes that successful development and regulatory approvals for these candidates could open new revenue streams and reinforce leadership in genetic therapy.

• Expense and margin management: While management anticipates increased R&D and SG&A expenses in the second half of the year due to integration of Inozyme and expanded clinical programs, they project continued revenue growth and operating margin discipline. Risks include timing of large orders, integration challenges, and the competitive landscape in rare disease therapeutics.

Catalysts in Upcoming Quarters

In the coming quarters, the StockStory team will monitor (1) the pace of VOXZOGO’s expansion in new indications and countries, (2) progress on pivotal trials for BMN 333 and BMN 401, and (3) integration of Inozyme’s pipeline assets into BioMarin’s commercial and R&D operations. Success in executing these initiatives, alongside maintaining adherence and supporting patients in rare disease markets, will be important markers of BioMarin’s progress.

BioMarin Pharmaceutical currently trades at $56.78, down from $60.35 just before the earnings. At this price, is it a buy or sell? The answer lies in our full research report (it’s free).

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