BioMarin Pharmaceutical (NASDAQ:BMRN) executives highlighted record 2025 revenue, expanding profitability, and multiple upcoming pipeline and regulatory milestones during the company’s fourth-quarter and full-year 2025 earnings call, while also outlining initial 2026 guidance that excludes contributions from the pending Amicus acquisition.
2025 results: record revenue growth led by Voxzogo and enzyme therapies
CEO Alexander Hardy said BioMarin delivered on its 2025 strategic goals while producing “outstanding growth.” Total 2025 revenue increased 13% to a record $3.22 billion, which Hardy attributed to a 9% increase in enzyme therapies revenue and a 26% rise in Voxzogo revenue.
Mueller also pointed to performance within the enzyme therapy portfolio, noting that Palynziq revenue increased 25% in Q4 and that it marked the fourth consecutive quarter with 20%+ year-over-year growth. For the full year, enzyme therapy revenue increased 9% year over year, led by 22% growth for Palynziq and 7% growth for Vimizim.
Voxzogo revenue totaled $927 million in 2025, up 26% versus 2024. Mueller said approximately 73% (nearly $680 million) of 2025 Voxzogo revenue was generated outside the U.S., which he said reflects the company’s global commercial reach.
Order timing and Q4 benefits; Roctavian withdrawal and charges
Mueller cautioned that fourth-quarter revenue benefited from order timing. He said BioMarin recognized revenue from an approximately $30 million contracted government order for Voxzogo that is not expected to repeat in Q1 2026. He also said stocking levels increased in the U.S. and select global markets for Voxzogo, Palynziq, and Vimizim during Q4.
On Roctavian, Mueller said BioMarin made a “strategic decision to withdraw it from the market.” As a result, the company recorded approximately $240 million in special items on a GAAP basis in the fourth quarter, with about half related to an inventory write-off that is not adjusted out of non-GAAP income.
BioMarin reported full-year 2025 non-GAAP diluted EPS of $3.15. Mueller added that excluding the 2025 IPRD and Roctavian charges included in non-GAAP income, underlying business EPS grew approximately 34%. Operating cash flow was $828 million in 2025, up 45% versus 2024.
2026 guidance (excluding Amicus) and financing update
BioMarin provided initial 2026 guidance excluding any post-close contributions from the announced Amicus acquisition, which management expects to close in the second quarter of 2026. Mueller said BioMarin will provide updated guidance for the combined business after closing and asked investors to wait for those details given that current Street models vary in whether they include the Amicus products.
For 2026, BioMarin guided to:
- Enzyme therapies revenue: $2.225 billion to $2.275 billion
- Voxzogo revenue: $975 million to $1.025 billion
- Royalties, Kuvan, and Roctavian revenue: $100 million to $125 million (Mueller described this as a 3% headwind to total revenue growth versus 2025)
- Total revenue: $3.325 billion to $3.425 billion
- Non-GAAP diluted EPS: $4.95 to $5.15 (including about $0.25 per share of pre-close integration preparation costs and interest expense related to the Amicus transaction)
Mueller said BioMarin’s organic non-GAAP operating margin expectation without Amicus is approximately 40% in 2026, consistent with prior targets. He reiterated the company’s prior view that the Amicus acquisition will be modestly dilutive in 2026, which could push operating margin slightly below 40%.
He also outlined expected quarterly dynamics: Q1 2026 is expected to be the lowest total revenue quarter, with total revenue and Voxzogo revenue expected to be on par with Q1 2025. Management expects the majority of pre-close Amicus costs to affect Q1 EPS, making it the lowest EPS quarter of 2026. Similar to 2025, the company anticipates international order timing will weigh the first half and benefit the second half, with results weighted to Q4.
Separately, Mueller said BioMarin secured approximately $3.7 billion of debt financing in preparation for the Amicus transaction, adding that demand supported “favorable pricing across the capital structure.”
Commercial and regulatory priorities: Palynziq label expansion and Voxzogo growth strategy
Chief Commercial Officer Cristin Hubbard said enzyme therapies grew across every product in 2025, driven by new patient starts and adherence. She said Palynziq is expected to remain the primary growth driver within the enzyme portfolio in 2026, supported by an anticipated adolescent label expansion. The company cited a U.S. PDUFA target action date of February 28 and anticipated European approval later in 2026.
On Voxzogo, Hubbard said more than 5,000 children worldwide were being treated at the end of 2025. She described the growth strategy as multi-pronged, with emphasis on new starts across all ages, particularly in children under two years old. Hubbard said approximately half of new starts in the fourth quarter were in children under age 2. She also cited examples of rapid uptake in newly launched markets, including approximately 40% penetration within seven months in a recently launched Asia-Pacific country and roughly 70% penetration within 12 months in a mid-sized European country.
In response to questions about competitive dynamics and switching, Hubbard said market research and field interactions suggest limited immediate incentive for patients doing well on Voxzogo to switch, emphasizing the importance of long-term safety and efficacy data. R&D chief Greg Friberg added that management viewed recently released one-year data for an FGFR3 inhibitor as “generally comparable” to other CNP class effects at one year, while stressing that longer-term durability and safety data will be important.
Mueller said BioMarin’s 2026 Voxzogo guidance range reflects scenario planning, with the lower end assuming stronger competitive impact from a potential first competitor in 2027 and “guarded” assumptions around routine market access renegotiations. Hardy later characterized certain reimbursement processes in established markets as an opportunity to broaden access, noting that these can involve price resets alongside the potential to reach more patients.
Pipeline milestones: full approval filing for Voxzogo, BMN 333, hypochondroplasia, and BMN 351
Friberg said BioMarin is preparing to submit a full approval package for Voxzogo in achondroplasia, including final adult height and a broader set of health and wellness outcomes. He said the company has collected over 10,000 patient-years of safety data, including patients treated for more than 10 years, and highlighted complications and outcomes BioMarin plans to present, including foramen magnum stenosis, symptomatic spinal stenosis, proportionality, and measures of mobility, gait, and quality of life.
BioMarin also detailed its plans for BMN 333, a long-acting CNP therapy for achondroplasia. Friberg said the phase III portion of a combined phase II/III study is designed to enroll 60 patients per arm and has 90% power to detect a 50% increase in annualized growth velocity versus Voxzogo, which he translated to 2.25 centimeters per year over placebo. In Q&A, Friberg said the company will use a Bayesian approach in phase II and could modify the protocol based on the totality of data.
On hypochondroplasia, Friberg said Voxzogo phase III data are expected in the first half of the year and noted the study is designed around an effect size roughly equivalent to achondroplasia, while citing investigator-sponsored data suggesting a larger improvement. Hubbard said diagnosis is a key challenge in hypochondroplasia, describing pre-launch, non-promotional efforts focused on education and referral/testing guidelines. Mueller later said BioMarin expects submissions in the second half of the year, and that approval and launch could follow in 2027, with immediate revenue impact at that time.
Friberg also discussed BMN 351 for Duchenne muscular dystrophy, saying the program showed 5% mean absolute dystrophin expression at week 25 in the 9 mg/kg cohort, which BioMarin’s PK/PD model predicts could translate to 10% at steady state. He said BioMarin is enrolling a 12 mg/kg cohort and plans to share those results in the second half of the year, while full results for the 6 and 9 mg/kg cohorts will be presented at the Muscular Dystrophy Association meeting in March.
About BioMarin Pharmaceutical (NASDAQ:BMRN)
BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe.
The company’s commercial portfolio includes several approved therapies targeting inherited disorders.
