BMRN

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Data from an investigator-led study showed treatment with VOXZOGO resulted in sustained improvements in growth over three years in children with hypochondroplasia New Phase 1 results also presented for investigational BMN 333 support potential weekly dosing in children with achondroplasia SAN RAFAEL, Calif., June 16, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced new data from studies of VOXZOGO® (vosoritide) in children with hypochondroplasia and the investigational medicine BMN 333 in achondroplasia were presented at ENDO 2026, the Endocrine Society Annual Meeting, in Chicago.

BioMarin (BMRN) reported earnings 30 days ago. What's next for the stock?

BMRN's shares rise after Voxzogo hits the main goal in a late-stage hypochondroplasia study, supporting planned FDA filings in 2026.

Study met primary endpoint, exceeding expectations with a highly statistically significant improvement in annualized growth velocity (AGV, change from baseline) of 2.33 cm/yr compared to placebo at week 52, as well as statistically significant increases in standing height and height Z-score Study demonstrated statistically significant improvement in arm span at week 52 compared to placebo, a key measure linked to functional independence Safety findings consistent with the established profile in achondroplasia with no new safety signals identified Supplemental New Drug Application (sNDA) submission to U.S. Food and Drug Administration (FDA) planned for third quarter, followed by submissions to the European Medicines Agency (EMA) and other regional health authorities  Full data to be presented at an upcoming medical meeting  SAN RAFAEL, Calif., May 20, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 CANOPY-HCH-3 study evaluating treatment with VOXZOGO ® (vosoritide) in children with hypochondroplasia met its primary endpoint, demonstrating a statistically significant increase in the change from baseline at week 52 in annualized growth velocity (AGV) compared to placebo (LS mean difference +2.33 cm/yr, p

BioMarin shares fall after BMN 401 meets a biomarker goal but misses a key radiographic endpoint in a late-stage ENPP1 deficiency study.

ENERGY 3 study met 1 of 2 co-primary endpoints in children with ENPP1 deficiency Treatment with BMN 401 led to statistically significant increases in plasma inorganic pyrophosphate (PPi) concentration, one of the study's co-primary endpoints; however, no improvement was observed in Radiographic Global Impression of Change (RGI-C) scores, the study's other co-primary endpoint and an important clinical measure of change in rickets severity Company is evaluating data to determine next steps SAN RAFAEL, Calif., May 18, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced results from the pivotal Phase 3 ENERGY 3 trial evaluating BMN 401 in children aged 1-12 with ENPP1 deficiency, a rare, serious and progressive genetic condition.

BioMarin Pharmaceutical remains a "Strong Buy," driven by pipeline progress, strategic acquisitions, and robust revenue guidance. BMRN's acquisition of Amicus Therapeutics adds GALAFOLD and POMBILITI + OPFOLDA, boosting 2026 revenue guidance to $3.825–$3.925 billion and targeting 20% YoY growth. VOXZOGO expansion into hypochondroplasia and ongoing clinical trials represent major catalysts, with topline Phase 3 data expected in Q2 2026.

BMRN misses Q1 EPS estimates despite a revenue beat, as charges and higher costs weigh on profits, while new Amicus deal lifts the 2026 outlook.

BioMarin Pharmaceutical Inc. (BMRN) Q1 2026 Earnings Call Transcript