BioMarin Pharmaceutical Inc. logo BMRN - BioMarin Pharmaceutical Inc.

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| PRICE TARGET: $87.40 DETAILS
HIGH: $120.00
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MEDIAN: $84.00
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UPSIDE: 47.76%

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U.S. Food and Drug Administration Accepts BioMarin's Supplemental New Drug Application for Full Approval of VOXZOGO® (vosoritide) for Children With Achondroplasia

U.S. Food and Drug Administration Accepts BioMarin's Supplemental New Drug Application for Full Approval of VOXZOGO® (vosoritide) for Children With Achondroplasia

Application based on long-term safety and efficacy data from three ongoing studies, including adult height and additional clinical outcomes beyond linear growth, including body proportionality and arm span evaluated over long-term follow-up FDA PDUFA target action date of Feb. 28, 2027 SAN RAFAEL, Calif., July 13, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA) for VOXZOGO ® (vosoritide) for full approval in children with achondroplasia.

Jul 13, 2026 05:05 AM prnewswire.com
BioMarin Announces New Three-Year VOXZOGO® (vosoritide) Data in Hypochondroplasia and BMN 333 Early Results at ENDO 2026

BioMarin Announces New Three-Year VOXZOGO® (vosoritide) Data in Hypochondroplasia and BMN 333 Early Results at ENDO 2026

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.

Jun 16, 2026 05:05 AM prnewswire.com
BioMarin Announces Positive Phase 3 Pivotal Study Results for VOXZOGO® (vosoritide) in Children with Hypochondroplasia

BioMarin Announces Positive Phase 3 Pivotal Study Results for VOXZOGO® (vosoritide) in Children with Hypochondroplasia

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

May 20, 2026 12:20 PM prnewswire.com
Here Are Tuesday’s Top Wall Street Analyst Research Calls: American Tower, Citigroup, CrowdStrike, Fortinet, Hanover Insurance, Jazz Pharmaceuticals, Stubhub, X-Energy, and More

Here Are Tuesday’s Top Wall Street Analyst Research Calls: American Tower, Citigroup, CrowdStrike, Fortinet, Hanover Insurance, Jazz Pharmaceuticals, Stubhub, X-Energy, and More

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

May 19, 2026 04:04 AM 247wallst.com
BioMarin Provides Update on Phase 3 Trial for BMN 401 in Children Aged 1-12 With ENPP1 Deficiency

BioMarin Provides Update on Phase 3 Trial for BMN 401 in Children Aged 1-12 With ENPP1 Deficiency

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.

May 18, 2026 05:05 AM prnewswire.com
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

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

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

May 11, 2026 03:58 AM 247wallst.com
BioMarin: 'Strong Buy' On VOXZOGO Expansion And Amicus Acquisition

BioMarin: 'Strong Buy' On VOXZOGO Expansion And Amicus Acquisition

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.

May 06, 2026 11:48 AM seekingalpha.com

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