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Jun 18, 2026
The FDA has reversed its earlier rejection of UniQure's Huntington's disease therapy AMT-130, accepting existing trial data for accelerated review. The company plans to submit a marketing application in Q3 2026, ending a dispute over sham surgery requirements.
Jun 17, 2026
The FDA agreed uniQure's existing trial data could support an accelerated approval filing for Huntington's therapy AMT-130, a reversal from earlier positions. Meanwhile, UK-based Harness Therapeutics selected HRN001 as its lead experimental candidate, targeting the FAN1 protein to address somatic expansion of Huntington's-causing genetic repeats.
Apr 21, 2026
The FDA said uniQure’s Phase I/II data for Huntington’s disease gene therapy AMT-130 are not adequate for a marketing application. The agency recommended a new randomised, double-blind, sham surgery-controlled study.
Apr 19, 2026
UniQure shares fell sharply after FDA comments appeared to raise concerns about the invasive delivery method for Huntington's disease therapy AMT-130. The company had planned a 2026 BLA filing after Phase I/II data showed a 75% slowing of disease progression at 36 months.
Mar 21, 2026
uniQure disclosed the FDA will not accept Phase I/II data alone for AMT-130 approval, causing shares to drop 49%. The company held regulatory meetings and reported $622.5 million in cash while investors face an April 13 class action deadline.
Mar 07, 2026
The FDA has denied or discouraged at least eight drug applications in the past year, including treatments for rare diseases, raising concerns about regulatory consistency and the future of experimental therapies.
Mar 03, 2026
The FDA informed uniQure that Phase 1/2 study data for AMT-130 in Huntington's disease is insufficient to support a biologics license application, recommending a prospective, randomized, double-blind, sham surgery-controlled study instead.
Mar 03, 2026
uniQure reports Q4 2025 results as investors await clarity on AMT-130 Huntington's disease gene therapy following FDA's November indication that Phase I/II data unlikely to support biologics license application.
Mar 02, 2026
uniQure received FDA feedback stating current AMT-130 data unlikely to support accelerated approval for Huntington's disease, while securities fraud lawsuits challenge prior disclosures. Fresh Fabry disease trial data showed elevated enzyme activity but prompted a dosing pause for safety review.
Feb 18, 2026
uniQure N.V. faces a securities fraud class action lawsuit after disclosing the FDA no longer agreed its AMT-130 data was adequate for approval, causing a 49% stock decline. Investors have until April 13, 2026 to file lead plaintiff applications.
Feb 15, 2026
A securities fraud class action lawsuit has been filed against uniQure N.V. alleging material misstatements about its Huntington's disease gene therapy AMT-130 and FDA approval pathway between September and October 2025.
Feb 13, 2026
Multiple law firms have filed securities fraud class actions against uniQure N.V. after the FDA reversed its position on the company's AMT-130 Huntington's disease drug candidate, causing shares to plummet 49% in a single day.
