BioMarin Q2 2026 Revenues Up 20% to $990M; VOXZOGO Hypochondroplasia sNDA Filed

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported second quarter 2026 total revenues of $990 million, a 20% increase year-over-year, and raised full-year 2026 guidance for total revenues, VOXZOGO, and non-GAAP diluted earnings per share. The addition of GALAFOLD and POMBILITI + OPFOLDA, with cost synergies, is expected to accelerate revenue growth, non-GAAP diluted EPS accretion, non-GAAP operating margin expansion, and operating cash flow through the mid-2030s. Strong global demand led the company to increase full-year VOXZOGO revenue guidance to at least $1 billion in 2026.

BioMarin recently submitted a supplemental New Drug Application to the U.S. Food and Drug Administration for approval of VOXZOGO for the treatment of hypochondroplasia. If approved, VOXZOGO would be the first targeted therapy for hypochondroplasia, with a potential 2027 launch. The company plans to provide an update on the application status as part of its third quarter earnings update. In May, the Phase 3 CANOPY-HCH-3 study of VOXZOGO in children with hypochondroplasia met its primary endpoint, with a statistically significant increase in annualized growth velocity at week 52 versus placebo (LS mean difference +2.33 cm/yr, p<0.0001), along with significant improvements in standing height, height Z-score, and the key secondary endpoint of arm span. The full Phase 3 dataset will be shared in a late-breaking oral presentation at the European Society for Paediatric Endocrinology Annual Meeting in September. At the Endocrine Society Annual Meeting in June, a Phase 2 investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia showed sustained improvements in growth with a favorable safety profile; mean annualized growth velocity increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p<0.001) and remained above baseline through year three, with mean height standard deviation score improving 0.72 over the three years.

Also at ENDO 2026, Phase 1 data for BMN 333, BioMarin's long-acting C-type natriuretic peptide for achondroplasia, demonstrated sustained exposure supporting weekly dosing and was well tolerated, with free CNP exposure at the highest dose more than 13-fold that of another long-acting CNP agent. The Phase 2/3 study is enrolling, with a data update expected in 2027. In July, the FDA accepted BioMarin's supplemental new drug application for full approval of VOXZOGO in children with achondroplasia, with a Prescription Drug User Fee Act target action date of February 28, 2027. In the second quarter, the European Commission approved PALYNZIQ for adolescents 12 years and older with phenylketonuria. PALYNZIQ is the only therapy that enables people with PKU to reach physiologic Phe levels while reducing dietary restrictions, regardless of severity.

During the quarter, BioMarin added BMN 820 (formerly DMX-200), a first-in-class oral CCR2 inhibitor for focal segmental glomerulosclerosis for which it holds exclusive U.S. commercialization rights. BMN 820 has the potential to treat a broad FSGS population, regardless of nephrotic syndrome status, and represents a U.S. total addressable patient population of approximately 30,000. The Phase 3 ACTION 3 trial is ongoing, with pivotal data expected in 2028. BMN 351, BioMarin's Phase 1/2 candidate for Duchenne muscular dystrophy, continued in development, and the company expects to provide a program update by year-end.

For the first quarter ended March 31, 2026, BioMarin reported total revenues of $766 million, an increase year-over-year. The company increased full-year 2026 total revenues guidance to between $3.825 billion and $3.925 billion, representing an accelerated growth rate of 20% year-over-year at the midpoint, reflecting the addition of GALAFOLD and POMBILITI + OPFOLDA to its commercial portfolio following the completion of the Amicus Therapeutics acquisition. In February, the U.S. FDA approved PALYNZIQ for adolescents 12 years of age and older with phenylketonuria, following Phase 3 PEGASUS data and under a REMS program due to immune-mediated safety risks including anaphylaxis. In March, initial Phase 1/2 data for BMN 351 showed dose-dependent increases in dystrophin expression at Week 25 biopsy in both the 6 and 9 mg/kg dose cohorts. In April, the first patient was enrolled in the registration-enabling Phase 2/3 study of BMN 333, and BioMarin submitted its U.S. supplemental new drug application for full approval of VOXZOGO for achondroplasia. Long-term VOXZOGO data presented at the Pediatric Endocrine Society annual meeting showed that children who initiated VOXZOGO treatment after age 5 achieved mean height gains of +10.60 cm after six years and +13.59 cm (p<0.0001 for both) after eight years of treatment, as compared to natural history data.