BioMarin raises 2026 revenue and VOXZOGO guidance, discontinues BMN 401

BioMarin Pharmaceutical reported second quarter 2026 results on August 6, 2026, with total revenues increasing 20% year-over-year to $990 million¹. The company raised its full-year outlook, citing strong first-half 2026 performance and second-half expectations for both Metabolic Conditions and VOXZOGO¹.

On the pipeline, BioMarin submitted a supplemental New Drug Application to the FDA for VOXZOGO in hypochondroplasia, which if approved would be the first targeted therapy for the condition, with a potential 2027 launch¹. That followed May data in which the Phase 3 CANOPY-HCH-3 study met its primary endpoint, showing 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 improvements in standing height, height Z-score, and arm span¹. Separately, the FDA in July accepted BioMarin's sNDA for full approval of VOXZOGO in achondroplasia, with a PDUFA target action date of February 28, 2027¹.

The company disclosed a significant pipeline decision: following the pivotal ENERGY 3 trial results announced in May, in which BMN 401 did not meet one of its two co-primary endpoints for ENPP1 deficiency, BioMarin has now made the decision to discontinue development of BMN 401 across all indications¹.

On guidance, strong U.S. and global demand led to increased full-year 2026 VOXZOGO revenue guidance to a low end of $1 billion¹, and updated total revenue guidance moved to $3,875 to $3,925 million, a 21% midpoint growth¹.

On cash, the balance sheet showed cash and cash equivalents of $874,005 thousand as of June 30, 2026, down from $1,311,679 thousand at December 31, 2025¹, reflecting $5,067,630 thousand used for the acquisition of Amicus, net of cash acquired¹ during the period.