BioMarin Lowers 2026 Outlook Despite Record Q3 Revenue

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) lowered its full-year 2026 revenue and non-GAAP earnings ranges after reporting record quarterly revenue, citing uncertainty over the timing of several orders from a single international customer and costs associated with recent pipeline investments. Third-quarter revenue surpassed $1 billion for the first time, supported by products acquired with Amicus Therapeutics and continued growth in VOXZOGO.

Total revenue rose 32% year over year to $1.025 billion in the quarter ended September 2026. Metabolic Conditions revenue increased 44% to $744 million, while VOXZOGO revenue grew 12% to $245 million despite a large international order shifting from the third quarter to the fourth.

BioMarin reported a GAAP diluted loss of $0.63 per share and a GAAP operating margin of negative 8.0%. Acquired in-process research and development expenses reached $288 million, primarily related to the acquisition of Alesta Therapeutics. On a non-GAAP basis, diluted earnings increased 92% to $0.23 per share, while operating margin expanded 10.6 percentage points to 13.4% as revenue growth outpaced underlying spending.

Metabolic portfolio drives growth

The first full quarter of contributions from GALAFOLD and POMBILITI + OPFOLDA was the main driver of the Metabolic Conditions portfolio’s growth. GALAFOLD generated $152 million of revenue, with patient numbers increasing 13% from a year earlier. BioMarin said expanded diagnosis and treatment, deeper penetration in existing and newer markets, greater engagement with key opinion leaders and family cascade testing supported growth.

POMBILITI + OPFOLDA recorded $49 million in quarterly revenue, while the number of patients receiving the therapy rose 69% year over year. Switching from other treatments contributed to growth, supported by clinical and real-world evidence, including five-year PROPEL data. New patient starts also benefited from supply constraints affecting other Pompe disease therapies.

Patients on therapy increased across all BioMarin-marketed Metabolic Conditions products. PALYNZIQ patient numbers rose 9%, led by its adolescent launch in the United States. VIMIZIM revenue advanced 18%, while NAGLAZYME revenue declined 11%, with both movements reflecting international order timing.

BioMarin said the commercial integration of Amicus continued, with 2026 cost-synergy realization tracking ahead of its expectations. The company expects the planned reductions—approximately $280 million on a GAAP basis and $220 million on a non-GAAP basis—to be fully realized in 2028.

VOXZOGO expands internationally

VOXZOGO revenue increased to $245 million from $218 million a year earlier, while the number of children treated globally rose about 17%. More than 75% of revenue came from outside the United States. In the U.S., children under age 2 accounted for most new patient starts.

The company is also preparing for a potential VOXZOGO launch in hypochondroplasia, a condition for which it estimates a total addressable patient population of about 14,000. The supplemental application has been accepted, with a May 30, 2027 PDUFA action date. The PDUFA date for full approval of VOXZOGO in achondroplasia is in February 2027.

Pipeline investment weighs on 2026 earnings outlook

BioMarin now expects 2026 total revenue of $3.80 billion to $3.90 billion, compared with its previous range of $3.875 billion to $3.925 billion. The midpoint represents approximately 20% year-over-year growth.

Metabolic Conditions revenue is projected at $2.675 billion to $2.75 billion, down from the prior $2.725 billion-to-$2.775 billion range. VOXZOGO revenue is expected to be $975 million to $1.025 billion, compared with the previous forecast of $1.00 billion to $1.05 billion. Other revenue, including KUVAN, ROCTAVIAN and royalties, is now forecast at $125 million to $150 million, up from $100 million to $125 million.

The company reduced its full-year non-GAAP diluted EPS forecast to $2.80 to $3.10 from $4.90 to $5.10. BioMarin expects the Alesta acquisition to result in approximately $320 million of 2026 pre-tax acquired IPR&D charges and ongoing operating expenses, reducing both GAAP and non-GAAP diluted EPS by about $1.55. The BMN 450 license agreement is expected to generate an additional $83 million of pre-tax charges and expenses, or an approximately $0.32-per-share impact.

BioMarin added four pipeline assets during 2026. These include BMN 450 for Fabry disease, BMN 440 for hypophosphatasia, BMN 430 for ReNU syndrome and BMN 820 for focal segmental glomerulosclerosis. A BMN 351 program update is expected by year-end, while data for BMN 333 and BMN 440 are anticipated in 2027. The adolescent indication for POMBILITI + OPFOLDA has a Jan. 10, 2027 PDUFA action date.

The outlook remains subject to the timing of international customer orders, successful integration and cost-synergy execution, clinical data readouts and regulatory decisions. BioMarin also noted that its non-GAAP measures exclude items including amortization, stock-based compensation and certain acquisition-related charges, and may not be directly comparable with similarly named measures used by other companies.