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BIOMARIN PHARMACEUTICAL INC (BMRN)·Q4 2024 Earnings Summary

Executive Summary

  • Q4 2024 revenue was $747 million (+16% y/y), driven by VOXZOGO and steady enzyme therapies; non-GAAP diluted EPS rose to $0.92 and non-GAAP operating margin expanded to 31.1% as cost transformation benefits flowed through .
  • 2025 guidance targets $3.1–$3.2B revenue, 32–33% non-GAAP operating margin, and $4.20–$4.40 non-GAAP EPS; VOXZOGO is expected to contribute $900–$950M, with growth weighted to H2 2025 .
  • VOXZOGO Q4 revenue reached $208M (26% U.S./74% OUS), while enzyme therapies rose 9% y/y; management emphasized early-treatment momentum in the U.S. 0–5 cohort and broader OUS penetration across 47 contributing countries .
  • Consensus estimate comparisons were unavailable at the time of writing due to S&P Global access limits; use actuals below for trajectory and margin analysis (values from company documents) .

What Went Well and What Went Wrong

What Went Well

  • Margin and EPS leverage: Q4 non-GAAP operating margin hit 31.1% (+1,340 bps y/y), with non-GAAP diluted EPS at $0.92 (+88% y/y), reflecting revenue mix, discontinued programs, and cost initiatives .
  • VOXZOGO expansion: Q4 VOXZOGO revenue was $208M (+42% y/y), with strong OUS mix (74%) and early-treatment focus in U.S. infants/young children; management noted published proportionality and guideline support for earlier therapy starts .
  • Cash generation and efficiency: FY 2024 operating cash flow reached $573M (+260% y/y), supporting reinvestment and BD optionality under the $500M cost transformation program .

Quotes:

  • “2024 was a year of record growth and profitability… non-GAAP diluted earnings per share increased 69%” .
  • “Fourth quarter non-GAAP operating margin of 31.1% was boosted by cost transformation… and strong revenue growth” .
  • “In the U.S., the majority of new patient starts… were for infants and young children under 5 years of age” .

What Went Wrong

  • SG&A headwinds: Higher SG&A due to increased bad debt expense tempered some operating leverage in the quarter .
  • Growth normalization pockets: Management flagged lower growth in 2025 for NAGLAZYME and ALDURAZYME given 2024 order timing bulges; overall FY25 growth profile is H2-weighted .
  • Competitive and scale implications: VOXZOGO growth rates decelerate on a larger base; management modeled competition and expects “start-and-stay” stickiness but acknowledged growth moderating as scale increases .

Financial Results

MetricQ4 2023Q2 2024Q3 2024Q4 2024
Revenue ($USD Millions)$646 $712.0 $746 $747
GAAP Diluted EPS ($)$0.11 $0.55 $0.55 $0.64
Non-GAAP Diluted EPS ($)$0.49 $0.96 $0.91 $0.92
GAAP Operating Margin %4.2% 16.9% 15.3% 21.6%
Non-GAAP Operating Margin %17.2% 31.2% 27.7% 31.1%
Consensus Revenue ($)Unavailable (S&P Global limit)Unavailable (S&P Global limit)Unavailable (S&P Global limit)Unavailable (S&P Global limit)
Consensus EPS ($)Unavailable (S&P Global limit)Unavailable (S&P Global limit)Unavailable (S&P Global limit)Unavailable (S&P Global limit)

Note: Consensus estimates unavailable at time of writing due to S&P Global API limits; values above are company-reported.

Segment/Product Revenues ($USD Millions)

ProductQ2 2024Q3 2024Q4 2024
VOXZOGO$183.9 $190 $208
VIMIZIM$178.0 $178 $191
NAGLAZYME$132.0 $132 $110
PALYNZIQ$88.3 $91 $100
BRINEURA$45.3 $37 $48
ALDURAZYME$38.6 $71 $39
KUVAN$28.6 $28 $28
ROCTAVIAN$7.4 $7 $11

Key KPIs

KPIQ2 2024Q3 2024Q4 2024
VOXZOGO patients on therapy (approx.)~3,500 ~3,800 N/A (mix provided)
VOXZOGO geo mix (U.S./OUS)N/AN/A26%/74% (Q4 2024); FY 2024 24%/76%
Operating Cash Flow ($USD Millions, FY)N/AN/A$573 FY 2024
Cash & Investments ($USD Millions)$1,781 (6/30/24) $1,492 (9/30/24) ~$1,659 (12/31/24)
VOXZOGO compliance (global avg.)N/AN/A~95% (management commentary)

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Total Revenues ($)FY 2024$2,750–$2,825 (Aug 5) $2,790–$2,825 (Oct 29) Raised at low-end
Non-GAAP Operating Margin %FY 202426–27% (Aug 5) 26.5–27.5% (Oct 29) Raised midpoint
Non-GAAP Diluted EPS ($)FY 2024$3.10–$3.25 (Aug 5) $3.25–$3.35 (Oct 29) Raised
Total Revenues ($)FY 2025N/A$3,100–$3,200 New
Non-GAAP Operating Margin %FY 2025N/A32–33% New
Non-GAAP Diluted EPS ($)FY 2025N/A$4.20–$4.40 New
VOXZOGO Contribution ($)FY 2025N/A$900–$950 New

Earnings Call Themes & Trends

TopicPrevious Mentions (Q2 & Q3)Current Period (Q4)Trend
VOXZOGO early-treatment focusQ2: Majority of U.S. starts in 0–5 cohort; ~3,500 patients; durable efficacy and safety Q4: Majority of U.S. starts 0–5; 26% U.S./74% OUS; guidelines endorse early treatment Continued expansion; earlier starts; OUS scale
Enzyme therapies growth & order timingQ2: Double-digit growth; NAGLAZYME order timing; ALDURAZYME supply dynamics Q4: 9% y/y growth; 2025 lower growth for NAGLAZYME/ALDURAZYME due to 2024 anomalies Moderation off 2024 bolus
Cost transformation & marginQ3: Raised FY24 guide; margin expansion; retired converts in cash Q4: Non-GAAP OM 31.1%; FY25 OM 32–33%; path to 40% in 2026 Executing; leverage visible
Business development (BD)Q3: Strategy updates, focus on ROCtavian rationalization Q4: 155 JPM meetings; preclinical & clinical assets targeted to sustain growth Increased BD priority
Pipeline progress (BMN333/351/349)Q2: BMN333 entering clinic 2025; BMN351 proof-of-concept expected 2025; BMN349 first-in-human later 2024 Q4: BMN333 dosing begun; BMN351 25-week data H2 2025; BMN349 MAD ongoing Advancing per plan
Regulatory/legal (IP)N/AIP action in EU vs Ascendis; decision expected in 12–15 months; defend IP in U.S. if needed Active defense posture
ROCTAVIAN strategyQ2: Focus U.S./Germany/Italy; profitable by end-2025 Q4: Modest revenue; strategy unchanged (implied) Focused roll-out, efficiency

Management Commentary

  • Strategy and performance: “Full year 2024 exceeded market expectations… top line grew 18%… non-GAAP diluted earnings per share increased 69%” .
  • Margin trajectory: “We are guiding [FY25] non-GAAP operating margin to 32–33%… stepping stone toward… 40% next year” .
  • Commercial drivers: “Record VOXZOGO results… majority of new patient starts in the U.S. were… under 5 years… we’re investing in commercialization to broaden reach” .
  • R&D priorities: “BMN333 PK study underway… BMN351 25-week biopsy data in H2 2025… CANOPY hypochondroplasia Phase 3 on track” .

Q&A Highlights

  • VOXZOGO growth cadence and geography: Management expects ~$200M y/y increase in VOXZOGO 2025 on a larger base; growth driven by U.S., Germany, Brazil; competition modeled with high “start-and-stay” stickiness .
  • H2 weighting: Overall FY25 growth skewed to Q3–Q4 across VOXZOGO and enzyme therapies as initiatives ramp .
  • Pipeline clarity: BMN333 aims for sustained free CNP exposures; BMN351 25-week biopsies provide line-of-sight to 10% dystrophin at steady state; BMN349 MAD enrolling with potential PD readouts .
  • Diagnostics and adherence: Enzyme therapy demand supported by gene panels and cascade screening (e.g., Brazil); PALYNZIQ growth driven by reinitiation and adherence programs in U.S./Japan .
  • IP stance: European Unified Patent Court action underway; U.S. enforcement if infringement observed .

Estimates Context

  • S&P Global consensus estimates for revenue and EPS were unavailable due to API request limits at time of writing; thus, formal beat/miss vs Street cannot be assessed here. Use company actuals and guidance trajectory for decision-making .

Key Takeaways for Investors

  • Margin leverage is intact: Q4 non-GAAP OM 31.1% and FY25 guide to 32–33% suggest continued cost program execution and operating scale, with path articulated to 40% in 2026 .
  • VOXZOGO remains the core growth engine: Early-treatment momentum and OUS penetration underpin VOXZOGO’s ~$900–$950M FY25 contribution; watch U.S. prescriber base expansion and OUS reimbursement/access additions .
  • Enzyme therapies durable but normalizing: Expect flatter NAGLAZYME/ALDURAZYME growth in FY25 given 2024 timing benefits; PALYNZIQ remains the key double-digit growth driver in the franchise .
  • 2025 growth back-half weighted: Commercial and diagnostic initiatives, plus BD optionality, point to stronger H2 prints; near-term trading likely to key off quarterly cadence confirmation .
  • Pipeline catalysts in 2025: BMN351 25-week biopsy data (H2), BMN333 PK (H2), PALYNZIQ adolescent Phase 3 midyear and potential sBLA/ EU filings in H2—each can shift medium-term revenue visibility .
  • Cash generation supports optionality: $573M FY24 operating cash flow and ~$$1.66B cash/investments provide flexibility for BD while funding priority programs .
  • Competitive narratives: Management expects VOXZOGO stickiness and safety profile to be a differentiator; monitor competitor timelines and any switching signals across geographies .

Appendix: Additional Data (Selected)

  • Q4 2024 product totals: Enzyme therapies $488M (+9% y/y), VOXZOGO $208M (+42% y/y), ROCTAVIAN $11M, KUVAN $28M (continued LOE impact) .
  • FY 2024 profitability: GAAP OM 17.0% (+930 bps y/y), non-GAAP OM 28.6% (+920 bps y/y); GAAP diluted EPS $2.21; non-GAAP diluted EPS $3.52 .
  • Balance sheet: 12/31/24 cash & equivalents $942.8M; cash & investments ~$1.659B; total assets $6.99B; total liabilities $1.33B; equity $5.66B .