UniQure said it will seek UK approval for its Huntington’s disease gene therapy AMT-130 after meeting with UK regulators and planning a marketing application later this year. The submission will be supported by three-year analyses from ongoing U.S. and European trials, which UniQure says show apparent disease-progression slowing at the highest dose. The update comes as UniQure awaits a second-quarter FDA meeting to discuss key elements of a potential Phase 3 trial design after the FDA demanded a new sham-controlled study approach, leaving the U.S. path uncertain. UniQure CEO Matt Kapusta framed the UK filing as a milestone for the Huntington’s community. Separately, the company signaled it expects feedback from the FDA tied to a four-year study analysis arriving in the third quarter, and it is also exploring potential approvals in additional countries.
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