uniQure’s stock has dropped by more than 40% after being asked to conduct another study of its Huntington’s disease gene therapy.
During a Type A meeting, the US Food and Drug Administration (FDA) told the company it does not agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130.
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As a result, the FDA has “strongly recommended” uniQure conduct a prospective, randomised, double-blind, sham surgery-controlled study.
uniQure will continue engaging with the FDA on the Phase III development and plans to request a Type B meeting in Q2 2026 to further discuss potential study design approaches.
The company’s stock, listed on the Nasdaq exchange, dropped 41% on the news, from a 27 February close of $15.63 to a 2 March open of $9.19. The company has a market cap of $613.6m.
Matt Kapusta, CEO of uniQure, said: “While we did not reach alignment on a submission pathway based on the Phase I/II data, we believe the totality and durability of our data warrant continued substantive dialogue regarding how the FDA’s stated commitment to regulatory flexibility may be appropriately applied in this setting.
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By GlobalData“We remain committed to engaging with the FDA to determine a clear, scientifically grounded, and efficient path forward for AMT-130. We are deeply grateful for the resilience and support of the Huntington’s disease community and remain committed to standing with patients and their families as we advance this potentially transformative therapy for a community in need.”
This is just the latest setback for uniQure’s Huntington’s disease gene therapy, after the company announced in November 2025 that the FDA had declined to review the drug. This announcement also caused the company’s stock to crash, from $67.69 at close on 31 October to $23.05 at market open on 3 November.
The biotech had presented positive data from the Phase I/II trial (NCT05243017) of its therapy in September 2025, with the trial having met its primary endpoint. Upon its potential approval, analysts at GlobalData forecast that the drug will make $1.31bn for uniQure in 2031.
There are currently no approved therapies for Huntington’s disease; however, in Australia, Skyhawk Therapeutics is hoping to gain approval of its small molecule therapy SKY-0515, which has been submitted to Australia’s Therapeutic Goods Administration (TGA).
uniQure is also facing challenges with its Fabry disease programme, with the FDA having ordered the company to pause dosing in February 2026 over safety concerns.
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