BioMarin (BMRN) Q4 2025 earnings review
ROCTAVIAN Capitulation Clears the Deck, But VOXZOGO Growth Hits a Wall
BioMarin delivered a strong Q4 top-line beat with revenues up 17% YoY, but the headline numbers are overshadowed by two massive narrative shifts. First, management finally killed the failed ROCTAVIAN gene therapy program, taking a brutal $240M charge that pushed GAAP operating margins negative. Second, despite VOXZOGO surging 31% in Q4, FY26 guidance implies a sudden and severe deceleration to just ~8% growth. The perfectly timed Amicus Therapeutics acquisition (closing Q2 2026) will provide a much-needed revenue lifeline, but organic growth for the company's former star asset is cooling rapidly.
🐂 Bull Case
By withdrawing ROCTAVIAN and absorbing the $240M hit now, BioMarin stops the cash burn from a failed asset. This paves the way for the guided 40% Non-GAAP operating margin in 2026.
The pending acquisition of Amicus Therapeutics will immediately diversify the portfolio with Galafold and Pombiliti, accelerating top-line growth and reducing reliance on VOXZOGO.
🐻 Bear Case
Guiding for ~8% VOXZOGO growth in FY26 after achieving 26% in FY25 confirms the competitive fears management signaled in Q3. The days of hyper-growth for this asset appear to be ending.
Non-GAAP Income for Q4 was heavily impacted by a $119M ROCTAVIAN inventory write-off hidden within Cost of Sales, muddying the true underlying operational profitability for the year.
⚖️ Verdict: ⚪
Neutral. The core Enzyme business is remarkably stable and the ROCTAVIAN write-off was necessary medicine. However, the abrupt deceleration in VOXZOGO guidance is a major concern that offsets the Q4 revenue beat.
Key Themes
ROCTAVIAN Capitulation Destroys Q4 Margins
After failing to find a buyer, BioMarin voluntarily withdrew ROCTAVIAN from the market. This triggered a massive $240M restructuring charge ($119M inventory write-off in COGS, $118M asset impairment in SG&A). Consequently, Q4 GAAP operating margin went Reversing, dropping to -5.1% from +21.6% a year ago.
VOXZOGO Growth Suddenly Braking
Q4 VOXZOGO sales were optically fantastic at $273M (+31% YoY), but management admitted this was driven by the timing of large Latin American government orders. The real story is the FY26 guidance: $975M-$1,025M. This midpoint implies Decelerating YoY growth of just 7.9%, a sheer drop from FY25's 26% growth, highlighting severe penetration limits or preemptive defensive positioning against upcoming competition.
Legacy Enzyme Portfolio Remains a Cash Cow
While VOXZOGO grabs the headlines, the Enzyme Therapies segment quietly delivered 13% YoY growth in Q4 ($549M). PALYNZIQ was the standout, Accelerating to 25% YoY growth ($125M). This durable, high-margin revenue base generated the cash needed to fund the Amicus acquisition.
Amicus Acquisition Masks Organic Weakness
The recently announced Amicus Therapeutics deal (closing Q2 2026) is a strategic masterstroke. By acquiring Galafold (Fabry Disease) and Pombiliti+Opfolda (Pompe Disease), BioMarin is buying high-margin revenue just as its organic VOXZOGO growth Decelerates. Management explicitly noted this will 'significantly accelerate and diversify revenues'.
Pipeline Pivot: BMN 333 & BMN 351 Advancing
Knowing VOXZOGO faces threats, management is rushing its successor, BMN 333 (long-acting CNP), into a registration-enabling Phase 2/3 study in 1H 2026. Separately, BMN 351 for Duchenne muscular dystrophy (DMD) showed 5.0% mean absolute dystrophin expression in early cohorts, with highly anticipated 12 mg/kg data due in 2H 2026.
KUVAN Generic Erosion Continues
KUVAN remains a structural drag on the top line. Q4 revenues Decelerated further, dropping 18% YoY to $23M due to relentless generic competition. Full-year KUVAN sales dropped 17% to $100M, confirming this asset will soon be immaterial to the broader business.
Other KPIs
Accelerating significantly from $572 million in FY24. This massive 45% jump in operating cash flow generation gave management the confidence and balance sheet firepower to execute the Amicus acquisition without straining liquidity.
Stable and strong. Up from $1.66 billion at the end of FY24. The company also secured $3.7 billion in non-convertible debt to fully fund the upcoming Amicus deal, maintaining structural flexibility.
Guidance
Decelerating. The midpoint of $3.375 billion implies just 4.8% YoY growth, a sharp slowdown from the 13% growth achieved in FY25. This excludes Amicus, laying bare the slowing organic momentum.
Decelerating slightly. The midpoint implies ~6.9% YoY growth, down mildly from the 9% growth posted in FY25, but still representing stable, predictable demand across the legacy portfolio.
Decelerating violently. Implies ~7.9% YoY growth at the midpoint, down from 26% in FY25. This suggests the "easy" patient capture phase is over, and geographic expansion is yielding diminishing marginal returns.
Accelerating on paper. Implies roughly 60% YoY growth vs FY25's $3.15. However, FY25 EPS was severely depressed by $1.10 in acquired IPR&D (Inozyme) and $0.46 in ROCTAVIAN inventory write-offs. Adjusting for those one-time hits, FY26 represents Stable, modest bottom-line growth.
Accelerating. Rebounding aggressively from 23.3% in FY25. The elimination of ROCTAVIAN OPEX and the lack of repeating IPR&D mega-charges makes this highly achievable.
Key Questions
VOXZOGO Growth Collapse
Your FY26 VOXZOGO guidance implies sub-8% growth. How much of this deceleration is driven by market saturation in the 0-4 age group versus preemptive pricing pressure or competitive share loss?
Amicus Integration and Margin Drag
You guided to a 40% organic Non-GAAP operating margin in 2026. Upon closing the Amicus deal in Q2, will the integration costs and Amicus's current margin profile dilute this 40% target for the back half of the year?
BMN 351 (DMD) Efficacy Bar
You reported 5.0% dystrophin expression for the 9 mg/kg cohort. With the market often demanding >10% for unambiguous clinical benefit, what is your minimum required threshold from the upcoming 12 mg/kg cohort to justify advancing to Phase 3?