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 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
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