FDA Priority Review for PALYNZIQ Adolescent Expansion Could Be a Game Changer for BioMarin (BMRN)

• BioMarin Pharmaceutical recently announced that the FDA has accepted its supplemental Biologics License Application for Priority Review to expand PALYNZIQ® treatment to adolescents aged 12–17 with phenylketonuria (PKU), setting a target action date for February 28, 2026.
• This regulatory milestone not only demonstrates ongoing progress in BioMarin's therapy pipeline but also signals potential growth in its rare disease treatment market.
• We'll look at how PALYNZIQ's regulatory advancement could impact BioMarin's investment narrative amid renewed interest in its pipeline.

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BioMarin Pharmaceutical Investment Narrative Recap

To be a shareholder in BioMarin Pharmaceutical, you need to believe in the company's ability to sustain and grow its leadership in rare disease therapies through ongoing pipeline innovation and successful market expansion of existing drugs like VOXZOGO and PALYNZIQ. The FDA’s acceptance of the PALYNZIQ priority review for adolescents enhances the company's near-term pipeline visibility, but does not materially change the biggest short-term catalyst, which remains international expansion and market share gains of VOXZOGO. The leading risk continues to be competition and pricing pressure in rare disease markets, with the latest PALYNZIQ news providing an incremental lift but not altering this central concern for BioMarin’s short-term outlook.

Among the recent announcements, BioMarin’s updated full-year 2025 earnings guidance, raising expected revenues to between US$3,150 million and US$3,200 million, offers relevant context. This comes as the PALYNZIQ label expansion advances, signaling management’s confidence in ongoing revenue streams from its core therapies, yet earnings visibility depends on successful execution against both competitive and regulatory hurdles. While incremental progress supports confidence in pipeline strength, investors continue to weigh anticipated expense growth tied to late-stage R&D and ongoing business development opportunities.

By contrast, there is a critical risk investors should be aware of relating to elevated spend on R&D and SG&A, especially if...

Read the full narrative on BioMarin Pharmaceutical (it's free!)

BioMarin Pharmaceutical's outlook anticipates $3.8 billion in revenue and $1.1 billion in earnings by 2028. This projection assumes a 7.6% annual revenue growth rate and a $442.8 million increase in earnings from the current $657.2 million.

Uncover how BioMarin Pharmaceutical's forecasts yield a $90.60 fair value, a 71% upside to its current price.

Exploring Other Perspectives

Simply Wall St Community members suggest fair value estimates for BioMarin that range widely from US$83.91 to US$151.18 across four analyses. Despite this diversity, ongoing expense growth connected to pipeline advancement and business development remains a key consideration for future profitability, so it pays to compare these perspectives before drawing conclusions.

Explore 4 other fair value estimates on BioMarin Pharmaceutical - why the stock might be worth just $83.91!

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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