Record Annual Revenue and Strong Top-Line Growth
Total 2025 revenues grew 13% year-over-year to a record $3.22 billion; Q4 2025 revenue was $875 million, up 17% YoY.
Voxzogo Rapid Expansion
Voxzogo revenue increased 26% for full year 2025 to $927 million and 31% in Q4; ~73% (~$680 million) of Voxzogo revenue was generated outside the U.S.; over 5,000 children treated worldwide by year-end.
Enzyme Therapies Durability and Growth
Full-year enzyme therapies revenue rose 9% in 2025 (Q4 +13% YoY), with the franchise described as a $2B+ business across an 80-country footprint.
Palynziq Outperformance and Regulatory Momentum
Palynziq revenue grew 22% for the year and 25% in Q4; an adolescent-label expansion has a U.S. PDUFA target action date of February 28, 2026, which management expects to drive additional uptake.
Profitability and Cash Flow Improvement
Reported full-year 2025 non-GAAP diluted EPS was $3.15; underlying business EPS (excluding IPR&D and Roctavian charges) grew approximately 34%; operating cash flow was $828 million, a 45% increase versus 2024.
2026 Guidance (Excluding Amicus Post-Close Contributions)
2026 guidance: enzyme therapies $2.225B–$2.275B; Voxzogo $975M–$1.025B; total revenues $3.325B–$3.425B; non-GAAP diluted EPS $4.95–$5.15; underlying organic operating margin ~40% (pre-Amicus).
Pipeline and R&D Milestones
Multiple near-term milestones: Phase III readouts planned for Voxzogo hypochondroplasia and BMN 401 (ENPP1 deficiency); BMN 333 Phase II/III initiation with Phase III powered (60 patients/arm) to detect a 50% increase in annualized growth velocity (2.25 cm/year over placebo); BMN 351 showed 5% mean absolute dystrophin at week 25 (9 mg/kg), predicting ~10% at steady state and additional cohorts enrolling.
Strategic Acquisitions and Financing
Inozyme acquisition adds BMN 401 (ENPP1 deficiency); Amicus acquisition (Galafold, Pombiliti, Opfolda) expected to close Q2 2026 and is projected to meaningfully uplift 2026+ revenues; secured approximately $3.7 billion of debt financing to support the Amicus transaction with favorable pricing.
Commercial Execution and International Penetration
Examples of commercial execution include ~40% penetration within 7 months in an Asia Pacific launch and ~70% penetration within 12 months in a midsized European country; management emphasizes focus on infants (0–2) and expanding in fast-growing ex-U.S. markets (≈75% of Voxzogo revenues ex-U.S.).