BioMarin Halts VOXZOGO Dosing in Select Phase 2 Trials
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BioMarin Pharmaceutical ( (BMRN) ) has issued an announcement.
On March 16, 2026, BioMarin announced it would discontinue dosing and enrollment in its Phase 2 VOXZOGO trials for Turner syndrome, SHOX-deficiency and aggrecan-deficiency after several slipped capital femoral epiphysis events were reported in two separate investigator-sponsored studies. The company emphasized that no such events have occurred in its own Phase 2 programs in these conditions, nor among more than 5,000 infants and children treated with VOXZOGO for achondroplasia over roughly 10,000 patient-years of safety data, and it will continue its Phase 2 CANOPY trials in Noonan syndrome and idiopathic short stature without aggrecan-deficiency, which account for the vast majority of participants in that study.
The move highlights BioMarin’s risk-management posture around VOXZOGO’s broader label expansion strategy while preserving development in larger potential markets such as idiopathic short stature. For stakeholders, the partial halt could temper expectations for some niche indications but suggests the company is prioritizing safety data and regulatory confidence, potentially supporting VOXZOGO’s longer-term positioning in pediatric growth disorders.
The most recent analyst rating on (BMRN) stock is a Buy with a $85.00 price target. To see the full list of analyst forecasts on BioMarin Pharmaceutical stock, see the BMRN Stock Forecast page.
Spark’s Take on BMRN Stock
According to Spark, TipRanks’ AI Analyst, BMRN is a Outperform.
The score is primarily supported by strong financial performance (multi-year revenue growth, high gross margins, strong cash generation, and a conservatively leveraged balance sheet) and a constructive earnings outlook with clear 2026 guidance. Technicals also support the score with price above major moving averages and positive momentum. The main constraint is valuation, with a higher P/E and no dividend yield indicated, alongside near-term headwinds and integration/dilution risks referenced in guidance.
To see Spark’s full report on BMRN stock, click here.
More about BioMarin Pharmaceutical
BioMarin Pharmaceutical Inc., a biotechnology company, develops and markets therapies for rare genetic diseases, including treatments targeting growth disorders in infants and children. Its portfolio includes VOXZOGO, used in achondroplasia and under investigation for additional short stature and genetic conditions, reflecting a strategic focus on niche, high-need pediatric indications.
Average Trading Volume: 2,468,045
Technical Sentiment Signal: Sell
Current Market Cap: $11.25B
For an in-depth examination of BMRN stock, go to TipRanks’ Overview page.
