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uniQure has previously announced that it held a Type A meeting with the U.S. Food and Drug Administration to discuss the Biologics License Application data package supporting accelerated approval of AMT-130, its investigational gene therapy for Huntington’s disease.
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This regulatory interaction is particularly important because it follows the FDA’s earlier view that existing Phase I/II AMT-130 data likely do not provide the primary evidence needed for a filing, leaving the development pathway and timelines under renewed scrutiny.
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We’ll now examine how this recent Type A FDA meeting on AMT-130’s data package could reshape uniQure’s investment narrative and risk profile.
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To own uniQure, you need to believe that AMT-130 can still become a first-in-class gene therapy for Huntington’s disease and ultimately support a viable commercial business despite ongoing losses. The recent Type A FDA meeting is central because it may redefine the path and timing for an AMT-130 filing, making regulatory clarity the key near term catalyst and extended regulatory uncertainty the single biggest risk to the story right now.
Among recent announcements, the FDA’s feedback that existing Phase I/II AMT-130 data likely do not provide the primary evidence needed for a BLA is the most relevant. It directly affects expectations for accelerated approval, interacts with uniQure’s dependence on AMT-130 as its main value driver, and raises the importance of whatever revised regulatory plan emerges from this Type A meeting as a focal point for upcoming catalysts.
Yet behind the promise of gene therapy for Huntington’s disease, investors should also be aware of the risk that…
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 $55.44 fair value, a 141% upside to its current price.
Six fair value estimates from the Simply Wall St Community span roughly US$14 to US$376 per share, reflecting very wide dispersion in expectations. Against this backdrop, the renewed uncertainty around AMT-130’s BLA path and timing underscores how differently future outcomes for uniQure’s business performance can be viewed, encouraging you to weigh several contrasting risk and catalyst scenarios.
