Biotech company BioMarin Pharmaceutical (NASDAQ: BMRN) missed Wall Street’s revenue expectations in Q1 CY2026 as sales rose 2.8% year on year to $766.2 million. On the other hand, the company’s full-year revenue guidance of $3.88 billion at the midpoint came in 1.2% above analysts’ estimates. Its non-GAAP profit of $0.76 per share was 16.9% below analysts’ consensus estimates.
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BioMarin Pharmaceutical (BMRN) Q1 CY2026 Highlights:
- Revenue: $766.2 million vs analyst estimates of $776 million (2.8% year-on-year growth, 1.3% miss)
- Adjusted EPS: $0.76 vs analyst expectations of $0.91 (16.9% miss)
- Adjusted EBITDA: $189.5 million vs analyst estimates of $150.8 million (24.7% margin, 25.6% beat)
- The company lifted its revenue guidance for the full year to $3.88 billion at the midpoint from $3.38 billion, a 14.8% increase
- Management lowered its full-year Adjusted EPS guidance to $4.95 at the midpoint, a 2% decrease
- Operating Margin: 16.9%, down from 30% in the same quarter last year
- Market Capitalization: $10.72 billion
StockStory’s Take
BioMarin’s first quarter saw revenue growth driven by continued demand for its enzyme therapies and Voxzogo, but the company missed Wall Street’s revenue and non-GAAP profit expectations. Management cited strong patient demand, especially for newly approved indications like adolescent Palynziq, but also acknowledged that order timing, elevated costs from manufacturing issues, and integration costs from the Amicus acquisition weighed on margins. CFO Brian Mueller described the quarter’s profitability as “significantly impacted” by these factors, noting that “the cost of sales charge and pre-close costs associated with the Amicus acquisition resulted in a $0.20 earnings per share impact.”
Looking forward, BioMarin’s updated guidance is anchored by the recent addition of Amicus’ therapies, which management expects will accelerate revenue growth and expand the company’s commercial reach. CEO Alexander Hardy emphasized that integration efforts are focused on leveraging BioMarin’s scale to boost diagnosis and treatment rates for newly acquired products. Management also highlighted upcoming pivotal data readouts for Voxzogo in hypochondroplasia and BMN-401 for ENPP1 deficiency as critical milestones, stating, “our immediate focus remains on the seamless and rapid integration of Amicus to accelerate our growth trajectory this year and beyond.”
Key Insights from Management’s Remarks
Management attributed Q1 results to steady demand for key therapies, the impact of recent label expansions, and the early effects of the Amicus acquisition, while noting that one-time costs and order timing affected reported margins and profit.
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Amicus acquisition integration: The addition of Galafold for Fabry disease and Pombility/Opfolda for Pompe disease is expected to accelerate revenue growth and diversify BioMarin’s portfolio. Management began targeted integration to leverage BioMarin’s scale in driving diagnosis and treatment rates.
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Voxzogo patient growth: New patient starts for Voxzogo, a therapy for achondroplasia, rose over 20% year over year, with notable uptake in children under age two. Management emphasized early diagnosis and treatment as key to sustained momentum, especially with a new competitor in the U.S. market.
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Palynziq adolescent launch: The recent label expansion of Palynziq to adolescents in the U.S. led to new patient enrollments and engagement from both experienced and new prescribers. Management expects this to support further growth as more patients titrate to their maintenance doses.
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Order timing and manufacturing costs: Reported revenue growth lagged underlying patient demand due to international order timing and U.S. stocking dynamics. A $31 million manufacturing charge and Amicus-related costs also pressured operating margin.
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Pipeline and regulatory milestones: Upcoming pivotal data for Voxzogo in hypochondroplasia and BMN-401 for ENPP1 deficiency are set for later in Q2, alongside ongoing progress in late-stage development programs like BMN-333 for skeletal disorders and BMN-351 for Duchenne muscular dystrophy.
Drivers of Future Performance
BioMarin’s forward outlook centers on integrating Amicus assets, maximizing uptake of expanded indications, and executing on near-term clinical milestones, while monitoring cost pressures and potential market disruptions.
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Amicus integration and product expansion: Management expects revenue growth to accelerate as Galafold, Pombility, and Opfolda are added to the commercial portfolio, with guidance implying high teens to low 20% organic growth for these products. Integration strategies target increased diagnosis rates in Fabry and switching in Pompe, supported by BioMarin’s global footprint.
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Key data readouts and regulatory progress: The company anticipates pivotal data for Voxzogo in hypochondroplasia and BMN-401 in ENPP1 deficiency in Q2, which could expand addressable markets if positive. Preparations for regulatory submissions and commercialization are underway, with a focus on identifying and diagnosing new patient populations early.
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Margin and profitability headwinds: Management highlighted that the Amicus acquisition will be slightly dilutive to non-GAAP earnings in 2026 due to upfront costs, with most profitability expected in the second half of the year. Additional risks include R&D investment, manufacturing costs, and regional market disruptions, particularly in the Middle East.
Catalysts in Upcoming Quarters
Going into the next quarters, the StockStory team will be watching (1) the pace of Amicus integration and initial revenue contributions from Galafold and Pombility/Opfolda, (2) pivotal clinical data for Voxzogo in hypochondroplasia and BMN-401 in ENPP1 deficiency, and (3) signs of margin improvement as one-time manufacturing and integration costs recede. Progress on regulatory submissions and the competitive landscape for skeletal condition therapies will also be important markers.
BioMarin Pharmaceutical currently trades at $55.00, in line with $55.46 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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