Skip to content
    BMRN
    Earnings call· Mar 2026(Q1 FY26)

    BIOMARIN PHARMACEUTICAL Q1 FY26 earnings call BMRN

    May 4, 2026 Source

    Executive summary

    BioMarin Q1 FY26 — Amicus Acquisition Closes, Raises FY26 Revenue Guidance

    BioMarin closed its acquisition of Amicus, significantly expanding its commercial portfolio with Galafold and Pombiliti/Opfolda, and raising its full-year revenue guidance. The company is focused on integrating the new assets to drive diagnosis and treatment rates, while also seeing strong demand for existing products like VOXZOGO and PALYNZIQ. Near-term pipeline catalysts include pivotal data readouts for VOXZOGO in hypochondroplasia and BMN 401 in ENPP1 deficiency.

    Highlights

    5

    • Amicus acquisition closed, accelerating anticipated FY26 revenue growth to 20% at the midpoint of updated guidance.

    • Total revenue guidance raised to $3.825 billion-$3.925 billion, with the midpoint representing 20% year-over-year growth.

    • Enzyme therapies revenue guidance raised to $2.725 billion-$2.775 billion, inclusive of Amicus products, resulting in approximately 30% growth at the midpoint.

    • VOXZOGO patient additions increased by more than 20% year-over-year globally, with over 50% of new U.S. patient starts from the under age 2 cohort in Q1.

    • PALYNZIQ adolescent label expansion launched successfully in the U.S., seeing encouraging early momentum and meaningful enrollments.

    Concerns

    4

    • Q1 non-GAAP diluted EPS was $0.76, significantly impacted by a $31 million charge for unsuccessful NAGLAZYME manufacturing process qualification and pre-close Amicus acquisition costs.

    • Q1 revenue was $766 million, impacted by U.S. order timing for PALYNZIQ and VOXZOGO, resulting in elevated stocking levels in Q4 2025.

    • Amicus acquisition expected to be slightly dilutive for full year 2026 non-GAAP diluted EPS.

    • Q2 non-GAAP diluted EPS expected to be only modestly higher than Q1, due to pre-close Amicus costs in April and higher 2026 Amicus dilution weighted to Q2.

    Guidance & targets

    7
    CategoryTargetConfidence
    Full-year 2026 Total Revenues
    $3.825B-$3.925B
    high materiality
    High
    Full-year 2026 Enzyme Therapies Revenue
    $2.725B-$2.775B
    high materiality
    High
    Full-year 2026 VOXZOGO Revenue
    $975M-$1.025B
    medium materiality
    High
    Full-year 2026 Non-GAAP Diluted Earnings Per Share
    $4.85-$5.05
    high materiality
    High
    VOXZOGO for hypochondroplasia approval
    potential approval
    medium materiality
    Medium
    2026 Total Revenues Recognition
    more than 55% in H2 2026
    medium materiality
    High
    2026 Earnings Per Share Recognition
    approximately 2/3 in H2 2026
    medium materiality
    High

    Segment performance

    3
    SegmentRevenueYoYQoQMargin
    Enzyme Therapies
    Led by growth in VIMIZIM, NAGLAZYME, and BRINEURA. PALYNZIQ first quarter revenues were impacted by U.S. order timing, resulting in elevated stocking levels in Q4 2025. Strong patient demand and adherence across the portfolio.
    VIMIZIM growthNAGLAZYME growthBRINEURA growthPALYNZIQ underlying patient base expansionPALYNZIQ adolescent label expansion momentum
    		—6%——
    VOXZOGO
    Strong patient demand with new patient starts across all regions and ages. Q1 results reflected expected order timing dynamics following a strong Q4 2025, particularly in international markets. Focus on driving new patient starts in children under 2 years of age.
    New patient starts across all regionsUnder age 2 cohort: >50% of new US patient starts in Q1Average age of initiation in under-2 segment: 10% decrease
    		—20% in patient additions globally——
    Amicus Products (Galafold, Pombiliti and Opfolda)
    Newly added to the portfolio following the Amicus acquisition. Expected to drive significant growth for enzyme therapies and total revenue. Initial priorities focus on driving diagnosis in Fabry and switching in Pompe, supporting increased penetration and geographic expansion.
    		Meaningful contributions———

    Operational metrics

    12
    Cost of sales charge
    $31M
    Q1 FY26

    Associated with an unsuccessful process qualification campaign to extend NAGLAZYME manufacturing capability. Did not impact commercial supply. Expected to be offset in full year 2026 non-GAAP diluted earnings per share guidance.

    Non-GAAP R&D expense
    increased year-over-year
    Q1 FY26

    Primarily due to spend to support BMN 401, VOXZOGO for hypochondroplasia, BMN 333, and BMN 351.

    Non-GAAP SG&A expense
    increased
    Q1 FY26

    Partially driven by investments to support commercial expansion across enzyme therapies and VOXZOGO and partially driven by pre-close costs associated with the Amicus acquisition.

    Amicus acquisition EPS impact
    $0.20
    Q1 FY26

    Impact to non-GAAP earnings per share result from cost of sales charge and pre-close costs associated with the Amicus acquisition.

    Amicus organic year-over-year growth rate
    high teens to low 20s
    FY26

    Implied by the updated guidance for Amicus products, based on full-year annual cycle comparison to $634 million reported for 2025.

    Hypochondroplasia identified patients
    90% increase
    current

    From diagnosis program and non-promotional pre-launch activities.

    Hypochondroplasia average age of diagnosis
    70% reduction
    current

    From diagnosis program and non-promotional pre-launch activities.

    VOXZOGO countries
    55
    to date

    Global expansion to date.

    BMN 333 AUC levels
    over 10x increases
    Phase I

    Achieved safely in healthy volunteers compared to baseline.

    BMN 351 dystrophin increases
    dose-dependent
    week 25

    Initial data presented at Muscular Dystrophy Association Congress.

    BMN 351 creatine kinase decreases
    notable decreases
    week 25

    Biomarker of muscle injury.

    BMN 351 functional decline prevention
    preventioncompared to historically matched controls
    week 25

    As measured by North Star Assessment and 6-minute walk test.

    Industry KPIs

    1
    MetricValueDetails
    Peak long term sales guidance$1B eachUSD

    Deals & partnerships

    1
    AmicusAcquisition of Amicus, adding Galafold for Fabry disease and Pombiliti and Opfolda for Pompe disease.

    Completed last week, starting a new and exciting chapter for BioMarin. Integration plan initiated, focused on leveraging BioMarin's operating scale and capabilities to drive diagnosis and treatment rates.

    Risks & headwinds

    4
    Geopolitical uncertainties in the Middle East2026

    modest amount of disruption

    Mitigation: Today's guidance reflects an allowance for this.

    NAGLAZYME manufacturing process qualification failureQ1 FY26

    $31M charge

    Mitigation: Did not impact commercial supply; expected to be offset in full year 2026 non-GAAP diluted EPS guidance.

    Order timing dynamics for PALYNZIQ and VOXZOGOQ1 FY26

    impacted Q1 revenues

    Mitigation: Expected to normalize, with year-over-year revenue growth for PALYNZIQ in full year 2026 and VOXZOGO revenue higher in H2 2026.

    Amicus acquisition dilutionFY26

    slightly dilutive for the full year 2026

    Mitigation: Expected to be accretive in the first 12 months after close and substantially accretive beginning in 2027.

    What to watch in Q2 FY26

    5

    Amicus integration roadmap and financial outlook

    Q2 call
    CurrentIntegration initiated, updated revenue guidance provided.
    TargetDetailed roadmap, commercial revenues, and cost synergies outlook.

    Why it matters

    Provides clarity on the long-term potential and financial impact of the Amicus acquisition, including peak sales and accretion.

    Next quarter, we plan to share this roadmap as well as more detail on BioMarin's growth acceleration with the addition of Galafold and Pombiliti and Opfolda to our commercial portfolio, and DMX200 for FSGS in Phase III to our late-stage pipeline.

    Q&A highlights

    9

    Contextualize bone mineral content benefits in hypochondroplasia data at PES; how does it inform expectations for top-line data?

    Greg Friberg explained that dexa scans presented at PES suggest healthy bone strength in addition to length, reiterating findings from achondroplasia. He expressed excitement for the Q2 Phase III top-line data, noting the strong scientific rationale and proof of concept from Dr. Dauber's investigator-sponsored study.

    “the related biology suggests again that the hypothesis is a strong one.”

    asked by Unknown Analyst · answered by Gregory Friberg

    2 min read6 chapters

    Detailed Narrative

    01

    Amicus Integration & Growth Strategy

    BioMarin completed the Amicus acquisition, adding Galafold for Fabry disease and Pombiliti/Opfolda for Pompe disease to its commercial portfolio. The integration plan focuses on leveraging BioMarin's operating scale and capabilities to drive diagnosis and treatment rates for these conditions. The company plans to share a detailed roadmap, including commercial and cost synergy outlook, on the next quarterly call. Management reiterated previous peak revenue potential estimates of approximately $1 billion each for Galafold and Pombiliti/Opfolda, with potential for these figures to be higher.

    02

    VOXZOGO Momentum & Expansion

    VOXZOGO continues to demonstrate strong patient demand, with new patient starts increasing across all regions. In the U.S., over half of new patient starts in Q1 were from children under age 2, and the average age of initiation in this segment decreased by 10%. The sNDA for full approval of VOXZOGO has been submitted to the FDA. The company is also preparing for potential expansion into hypochondroplasia, with Phase III data expected in Q2 2026 and potential approval in 2027, supported by pre-commercialization activities focused on diagnosis and awareness.

    03

    Enzyme Therapies Performance

    The enzyme therapies portfolio delivered 6% year-over-year growth in Q1, led by VIMIZIM, NAGLAZYME, and BRINEURA. PALYNZIQ saw encouraging early momentum following its adolescent label expansion in the U.S. in February, with meaningful enrollments and new patient starts in the under-18 age group. The company expects year-over-year revenue growth for PALYNZIQ in full year 2026 as these new patient starts gain momentum and patients titrate to their maintenance dose.

    04

    Pipeline Progress & Milestones

    BioMarin anticipates several key R&D milestones in 2026, including Phase III top-line results for VOXZOGO in hypochondroplasia and BMN 401 in ENPP1 deficiency, both expected in Q2. Enrollment is underway for the global registrational-enabling Phase II/III study of BMN 333, a long-acting CNP therapy for achondroplasia, aiming for a superiority profile. Initial data for BMN 351 in muscular dystrophy showed dose-dependent dystrophin increases and functional improvements, with enrollment ongoing in the 12 mg/kg cohort.

    05

    Financial Outlook & Timing

    Q1 2026 total revenues were $766 million, with non-GAAP diluted EPS of $0.76. These results were impacted by a $31 million charge for an unsuccessful NAGLAZYME manufacturing process qualification and pre-close Amicus acquisition costs. The company expects more than 55% of total 2026 revenues and approximately two-thirds of 2026 EPS to be recognized in the second half of the year, driven by order timing for historical products and the weighting of Amicus revenues and dilution.

    06

    Hypochondroplasia Market Preparation

    BioMarin is actively engaged in pre-launch activities for VOXZOGO in hypochondroplasia, focusing on driving diagnosis and awareness. These non-promotional efforts have already led to a 90% increase in identified hypochondroplasia patients and a 70% reduction in the average age of diagnosis. The goal is to ensure patients are identified and ready for therapy upon potential launch, accelerating the adoption curve.

    AI-generated summary of the company’s earnings call. Not investment advice.