BioMarin tops $1B a quarter on Amicus, but order timing and IPR&D charges cut 2026 guidance

The quarter in numbers

BioMarin reported third-quarter 2026 total revenues of $1,025 million, up 32% from $776 million a year earlier. The company said it was the first quarter with revenue above $1 billion. Nine-month revenue was $2,781 million, up 18% from $2,347 million.

Profitability moved in opposite directions depending on the measure. The GAAP net loss widened to $123 million from $31 million, or $(0.63) per diluted share against $(0.16). Non-GAAP income roughly doubled to $45 million from $22 million, with non-GAAP diluted EPS of $0.23 versus $0.12. GAAP operating margin was (8.0)% against (6.0)% a year ago, while non-GAAP operating margin rose to 13.4% from 2.8%.

What drove growth: Amicus products and Metabolic Conditions

Metabolic Conditions revenue rose 44% to $744 million, and 26% year to date. The company credited the acquired GALAFOLD and POMBILITI + OPFOLDA, plus higher VIMIZIM revenue. Both acquired products came with the Amicus deal, which closed April 27, 2026, so this was their first full quarter inside BioMarin. GALAFOLD contributed $152 million and POMBILITI + OPFOLDA $49 million. Patients treated rose approximately 13% and 69% Y/Y, respectively. The company said POMBILITI + OPFOLDA uptake benefited from supply constraints for other Pompe therapies.

Beyond the acquired assets, the picture was mixed. VIMIZIM grew 18% to $216 million, which the company attributed to the timing of large international orders. NAGLAZYME fell 11% to $108 million on the same timing factor. PALYNZIQ rose 2% to $111 million, BRINEURA 10% to $53 million and ALDURAZYME 6% to $57 million. KUVAN slipped to $23 million, and ROCTAVIAN, which the company said it will no longer market, recorded $1 million.

VOXZOGO and the order-timing problem

VOXZOGO revenue grew 12% to $245 million, which the company attributed to new patients starting therapy. The number of children treated globally rose 17% Y/Y, with patients in 58 countries on therapy. More than 75% of VOXZOGO revenue came from outside the U.S.; in the U.S., the majority of new patient starts were in children under two.

That international weighting is central to the guidance change. The company said a large international order expected in the third quarter is now anticipated in the fourth, and it updated guidance for Total Revenues, Metabolic Conditions and VOXZOGO to reflect uncertainty in the timing of multiple orders from a single international customer.

Guidance: revenue trimmed, EPS reset by acquired R&D

Full-year 2026 total revenue guidance is now $3,800 million to $3,900 million, down from $3,875 million to $3,925 million, implying 20% growth at the midpoint. Metabolic Conditions guidance is $2,675 million to $2,750 million (from $2,725 million to $2,775 million), and VOXZOGO is $975 million to $1,025 million (from $1,000 million to $1,050 million). The VOXZOGO midpoint implies 8% growth. Other Revenues, which include KUVAN, ROCTAVIAN and royalties, were raised to $125 million to $150 million from $100 million to $125 million.

The larger change is on earnings. Non-GAAP diluted EPS guidance falls to $2.80 to $3.10 from $4.90 to $5.10, a midpoint decline of 6% Y/Y, based on roughly 200 million weighted-average diluted shares. The company expects the Alesta acquisition and the BMN 450 license to produce total pre-tax charges of approximately $320 million and $83 million for 2026. These cover acquired IPR&D and ongoing operating expenses, with an unfavorable EPS impact of approximately $1.55 and $0.32, respectively, on both GAAP and non-GAAP EPS. Those two figures total about $1.87, while the guidance midpoint falls by $2.05, so the release implies other factors also weigh on the revised range without itemising them.

Costs, synergies and the Ascendis settlement

The company tied the wider GAAP loss to Amicus-related intangible amortization, interest expense on acquisition debt, amortization of inventory fair value step-up, integration and restructuring costs, and the Alesta IPR&D charge. Non-GAAP income was helped by revenue growth but offset by higher IPR&D, higher interest expense and increased sales and marketing spend behind GALAFOLD and POMBILITI + OPFOLDA. Year to date, the effect is clear: GAAP net income was $27 million versus $395 million, and non-GAAP EPS was $2.18 versus $2.69.

The company said it is realizing accelerated cost synergies and still expects approximately $280 million of GAAP cost synergies (about $220 million non-GAAP) to be fully realized in 2028. Separately, the August 30 global settlement with Ascendis over Yuviwel gives BioMarin a royalty of 20% of U.S. net sales, retroactive to first commercial sale, and 18% of net sales in the EU, Brazil and South Korea until May 2030.

Pipeline, regulatory catalysts and what to watch

The nearest regulatory dates are both VOXZOGO. FDA's PDUFA date for full approval in children with achondroplasia is February 28, 2027, and the supplemental NDA for hypochondroplasia has a May 30, 2027 date. The EMA has validated the HCH submission. Beyond that, the company expects a BMN 351 (Duchenne) program update by year-end, BMN 333 dose-response data and BMN 440 (HPP) Phase 1/2a data in 2027, and pivotal BMN 820 (FSGS) data in 2028. BMN 450, the new investigational subcutaneous Fabry ERT, is in Phase 1/2.

Near-term markers are whether the delayed international order lands in the fourth quarter, whether GALAFOLD and POMBILITI + OPFOLDA sustain their patient growth once Pompe supply constraints ease, and how quickly synergies offset the acquisition costs.