Will Strong Q2 Results and Five-Year ROCTAVIAN Data Shift BioMarin’s (BMRN) Rare Disease Narrative?

• BioMarin Pharmaceutical recently reported robust fiscal Q2 2025 results, highlighting meaningful year-over-year gains in revenue and earnings, alongside new long-term data showing five-year clinical benefit for its gene therapy ROCTAVIAN.
• Management also raised revenue guidance and will present at three major healthcare investor conferences in September, emphasizing momentum in the company's development pipeline and efforts to engage stakeholders on future growth prospects.
• We'll examine how BioMarin’s raised revenue outlook and long-term gene therapy data influence the company’s rare disease investment narrative.

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BioMarin Pharmaceutical Investment Narrative Recap

To own BioMarin Pharmaceutical stock, you need confidence in the company’s ability to drive revenue growth from its rare disease therapies while successfully launching new products and expanding internationally. The latest strong Q2 results and raised guidance reinforce near-term momentum, but do not significantly shift the main short-term catalyst: sustained commercial execution of core drugs like VOXZOGO. The biggest risk remains intensifying competition, particularly in rare disease markets, which could pressure future revenue and margin growth, and the news hasn’t altered that risk materially.

Among recent announcements, the five-year clinical data for ROCTAVIAN is especially relevant, offering tangible evidence of durable efficacy that could support future adoption and guide payer and regulatory discussions. Durable gene therapy results help defend BioMarin’s growth story and may provide a counterbalance to pressure from rivals, though ongoing competition and pricing uncertainty still weigh on the investment outlook.

However, investors should also keep in mind that, despite the promising news, risks around future pricing and reimbursement could...

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 7.7% yearly revenue growth and a $442.8 million earnings increase from $657.2 million today.

Uncover how BioMarin Pharmaceutical's forecasts yield a $97.62 fair value, a 68% upside to its current price.

Exploring Other Perspectives

Simply Wall St Community members submitted five fair value estimates for BioMarin, spanning US$64.55 to US$186.29 per share. While many anticipate expanding demand and clinical progress, persistent competition remains a factor shaping long-term performance and investor debate.

Explore 5 other fair value estimates on BioMarin Pharmaceutical - why the stock might be worth over 3x more than the current price!

Build Your Own BioMarin Pharmaceutical Narrative

Disagree with existing narratives? Create your own in under 3 minutes - extraordinary investment returns rarely come from following the herd.

• A great starting point for your BioMarin Pharmaceutical research is our analysis highlighting 3 key rewards 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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