BMRN's shares rise after Voxzogo hits the main goal in a late-stage hypochondroplasia study, supporting planned FDA filings in 2026.
BioMarin Pharmaceuticals Inc. (NASDAQ:BMRN) stock is trading higher on Thursday as the company is celebrating positive results from its Phase 3 CANOPY-HCH-3 study for VOXZOGO (vosoritide) in children with hypochondroplasia.
BioMarin Pharmaceutical said on Wednesday its treatment for a rare condition that leads to short stature helped boost growth in children, meeting the main goal of a late-stage study and sending shares up nearly 5% in extended trading.
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.
Pre-Market Stock Futures: Futures are trading lower on Tuesday as the sell-off in technology stocks carried through to Monday and is headed down that road today. All of the major indices, except the Dow Jones Industrial Average, which closed 0.32% higher at 49,668, finished the day lower. The small-cap Russell 2000 was the big loser... Here Are Tuesday's Top Wall Street Analyst Research Calls: American Tower, Citigroup, CrowdStrike, Fortinet, Hanover Insurance, Jazz Pharmaceuticals, Stubhub, X-Energy, and More
BioMarin Pharmaceutical said on Monday its experimental treatment for a rare genetic condition met one of the two main goals in a late-stage 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.
Pre-Market Stock Futures: Futures are trading mixed as we get set to start the new trading week, as reports indicate that President Trump declined Iran's counteroffer for peace. This comes after a remarkable Friday, when stocks roared to record highs, driven primarily by a stronger-than-expected April jobs report that eased economic concerns and by a... Here Are Monday's Top Wall Street Analyst Research Calls: Dell Technologies, BioMarin Pharmaceutical, Disney, HubSpot, Klarna, Oklo, Pitney Bowes, Trade Desk, Wendy's, and More
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.
