- Earlier in March 2026, BioMarin Pharmaceutical halted dosing and enrollment in Phase 2 VOXZOGO trials for Turner syndrome, SHOX-deficiency and ACAN-deficiency after slipped capital femoral epiphysis events emerged in separate investigator-sponsored studies, while allowing other VOXZOGO programs, including Noonan syndrome and most idiopathic short stature patients, to continue.
- Just days before the halt, BioMarin reported new data showing durable, multi-year growth and proportionality benefits for VOXZOGO in very young children with achondroplasia, underscoring a complex balance between expanding indications and managing safety signals across different patient groups.
- We’ll now examine how suspending certain VOXZOGO indications after slipped capital femoral epiphysis events might influence BioMarin’s rare-disease investment narrative.
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BioMarin Pharmaceutical Investment Narrative Recap
To own BioMarin, you need to be comfortable with a rare-disease story that leans heavily on VOXZOGO and ongoing label expansion. The recent halt in certain VOXZOGO Phase 2 indications highlights clinical and safety risk, but it does not directly touch the core achondroplasia franchise, which remains the key near term driver. The biggest risk right now is that any broader safety concerns around VOXZOGO could amplify portfolio concentration risk and pressure revenue and margins.
The March 12 VOXZOGO data in very young children with achondroplasia is particularly relevant here, because it reinforces multi year growth and proportionality benefits in the indication that currently underpins VOXZOGO’s commercial value. Those results, supported by more than 10,000 patient years of safety data and no slipped capital femoral epiphysis events in achondroplasia or hypochondroplasia trials, frame the latest trial suspensions as a targeted response rather than a wholesale reset of the VOXZOGO thesis.
Yet despite the reassuring achondroplasia data, investors should be aware that concentrated dependence on VOXZOGO leaves BioMarin especially exposed if...
Read the full narrative on BioMarin Pharmaceutical (it's free!)
BioMarin Pharmaceutical's narrative projects $3.8 billion revenue and $1.1 billion earnings by 2028. This requires 7.6% yearly revenue growth and about a $400 million earnings increase from $657.2 million today.
Uncover how BioMarin Pharmaceutical's forecasts yield a $88.96 fair value, a 62% upside to its current price.
Exploring Other Perspectives
Some of the most optimistic analysts were expecting revenue of about US$4.2 billion and earnings of US$1.4 billion by 2028, which assumes that VOXZOGO’s expansion and broader pipeline setbacks remain limited, yet the latest safety related pause shows how quickly clinical risk can challenge even the rosiest projections and why you should weigh several viewpoints before deciding what you believe.
Explore 6 other fair value estimates on BioMarin Pharmaceutical - why the stock might be worth just $64.55!
Form Your Own Verdict
Don't just follow the ticker - dig into the data and build a conviction that's truly your own.
- A great starting point for your BioMarin Pharmaceutical research is our analysis highlighting 2 key rewards and 2 important warning signs that could impact your investment decision.
- Our free BioMarin Pharmaceutical research report provides a comprehensive fundamental analysis summarized in a single visual - the Snowflake - making it easy to evaluate BioMarin Pharmaceutical's overall financial health at a glance.
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This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned.
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