AMT-130 is an investigational one-time AAV5 gene therapy designed to lower huntingtin expression in Huntington's disease. It is being evaluated in phase 1/2 clinical trials in adults with early manifest disease, with ongoing regulatory discussions on approval pathway requirements. It is not approved for marketing.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
