uniQure (QURE) Is Down 17.3% After FDA Questions AMT-130 Data Sufficiency For BLA - Has The Bull Case Changed?

• In late 2025, uniQure N.V. reported that the FDA’s final minutes from an October pre-BLA meeting indicated Phase I/II data for AMT-130 in Huntington’s disease are currently unlikely to support a Biologics License Application, prompting the company to seek a follow-up meeting in early 2026.
• This feedback introduces additional uncertainty around AMT-130’s regulatory path and timing, raising questions about one of uniQure’s most important pipeline drivers.
• We’ll now examine how the FDA’s concern over AMT-130’s Phase I/II data reshapes uniQure’s investment narrative and risk profile.

Find companies with promising cash flow potential yet trading below their fair value.

uniQure Investment Narrative Recap

To own uniQure today, you need to believe that AMT-130 can still translate its early Huntington’s disease data into a viable regulatory path and commercial opportunity, despite ongoing losses and funding needs. The FDA’s view that current Phase I/II data are unlikely to support a BLA removes a near term approval catalyst and amplifies the central risk that uniQure’s lead asset may require more evidence, time, and capital than previously hoped.

The December 2025 FDA meeting minutes directly undercut the earlier 2025 expectation of a potential Q1 2026 BLA filing that many investors watched as a key milestone. That shift, together with the substantial US$175,000,000 Hercules term loan and recent equity raise, makes future regulatory feedback on AMT-130 the focal catalyst for reassessing both the company’s risk profile and its ability to fund any additional trials.

Yet investors should also be aware that the company’s dependence on AMT-130 and uncertain FDA interactions could...

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

uniQure's narrative projects $306.4 million revenue and $32.3 million earnings by 2028. This requires 147.5% yearly revenue growth and a $249.9 million earnings increase from $-217.6 million today.

Uncover how uniQure's forecasts yield a $58.21 fair value, a 156% upside to its current price.

Exploring Other Perspectives

Six fair value estimates from the Simply Wall St Community span roughly US$13 to more than US$350 per share, reflecting sharply different expectations. When you weigh those views against the FDA’s reservations around AMT-130’s Phase I/II data, it underlines how critical future regulatory feedback could be for the company’s trajectory and why it is worth comparing multiple perspectives before forming an opinion.

Explore 6 other fair value estimates on uniQure - why the stock might be worth 40% less than the current price!

Build Your Own uniQure 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 uniQure research is our analysis highlighting 2 key rewards and 3 important warning signs that could impact your investment decision.
• Our free uniQure research report provides a comprehensive fundamental analysis summarized in a single visual - the Snowflake - making it easy to evaluate uniQure'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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