Ultragenyx Pharmaceutical (RARE) is back in focus after reporting preliminary 2025 revenue guidance of US$672 million to US$674 million, which is above its prior range, alongside completing an FDA BLA submission for gene therapy candidate DTX401.

The raised 2025 revenue guidance and DTX401 BLA completion come after a volatile period, with a 7 day share price return of 6.97% but a 30 day share price return of a 33.45% decline, and a 1 year total shareholder return of a 39.76% decline. Together, these figures suggest that recent momentum is tentatively rebuilding from a weak longer term base as investors reassess both growth potential and risk around Ultragenyx’s rare disease pipeline.

If this kind of pipeline driven story has your attention, it could be a useful moment to scan other healthcare stocks that might suit your watchlist next.

With the share price sharply lower over the past year and revenue guidance now upgraded to about US$673 million, the key question is whether Ultragenyx is still being discounted or if the market already reflects its future growth potential.

Most Popular Narrative: 70.5% Undervalued

With Ultragenyx shares at US$24.11 against a narrative fair value of US$81.85, the gap between price and expectations is wide and very clear.

Ultragenyx's clinical pipeline is advancing with five Phase III programs (including UX143 and GTX-102), multiple BLA submissions expected in the coming quarters, and near-term Phase III data readouts (notably for UX143 in OI by year-end and GTX-102 in Angelman syndrome in 2026) serving as upcoming value inflection points that can diversify and significantly accelerate the company's revenue base.

Want to understand why this growth story points to a much higher fair value? Revenue expansion, margin shifts and future earnings expectations all sit at the core of this narrative. Curious how those pieces fit together and what has to go right to justify that valuation gap?

Result: Fair Value of $81.85 (UNDERVALUED)

However, this optimistic setup still hinges on successful regulatory outcomes for key gene therapies and on Ultragenyx reducing cash burn to avoid further funding pressure or dilution.

Build Your Own Ultragenyx Pharmaceutical Narrative

If you see the story differently or prefer to analyze the numbers yourself, you can create a custom view in minutes with Do it your way.

A great starting point for your Ultragenyx Pharmaceutical research is our analysis highlighting 2 key rewards and 2 important warning signs that could impact your investment decision.

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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.