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    Ultragenyx Pharmaceutical (RARE) Is Down 31.0% After Rare-Disease Milestone Updates - Has The Bull Case Changed?

    Simply Wall St·01/04/2026 23:29:24
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    • Ultragenyx Pharmaceutical recently reported two key milestones: strong Phase 3 results for setrusumab (UX143) in Osteogenesis Imperfecta and completion of its Biologics License Application to the FDA for DTX401 gene therapy in Glycogen Storage Disease Type Ia, supported by up to six years of clinical follow-up data.
    • These advances highlight Ultragenyx’s push to convert late-stage rare disease programs into potential commercial therapies that could broaden its treatment portfolio and reinforce its focus on high unmet medical need.
    • We’ll now examine how the BLA completion for DTX401 and positive UX143 data may reshape Ultragenyx’s investment narrative and outlook.

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    Ultragenyx Pharmaceutical Investment Narrative Recap

    To own Ultragenyx, you need to believe its late stage rare disease pipeline can eventually offset persistent losses and a short cash runway. The BLA completion for DTX401 and positive UX143 Phase 3 data sharpen the near term regulatory catalysts, but they do not remove the core risk around ongoing cash burn and potential dilution.

    The most relevant recent announcement is the completed BLA submission for DTX401 in Glycogen Storage Disease Type Ia, backed by up to six years of follow up data and favorable Phase 3 GlucoGene outcomes on cornstarch dependence, glycemic control, and quality of life. This filing moves one of Ultragenyx’s key gene therapy assets closer to a potential approval decision, directly tying into the company’s reliance on regulatory success to broaden its revenue base.

    However, while these clinical and regulatory steps are encouraging, investors should still be aware of the company’s high cash burn and limited cash runway...

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

    Ultragenyx Pharmaceutical's narrative projects $1.4 billion revenue and $46.9 million earnings by 2028.

    Uncover how Ultragenyx Pharmaceutical's forecasts yield a $81.85 fair value, a 247% upside to its current price.

    Exploring Other Perspectives

    RARE 1-Year Stock Price Chart
    RARE 1-Year Stock Price Chart

    Two fair value estimates from the Simply Wall St Community span roughly US$82 to US$329 per share, showing how far apart individual expectations can be. Against that wide range, Ultragenyx’s dependence on successful regulatory outcomes for assets like DTX401 and UX111 becomes a central issue for how its future performance might evolve, so it is worth reviewing several different viewpoints before forming your own stance.

    Explore 2 other fair value estimates on Ultragenyx Pharmaceutical - why the stock might be worth just $81.85!

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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.