UniQure said it plans to seek UK approval for AMT-130, its Huntington’s gene therapy, after a positive meeting with UK regulators. The company plans a marketing application later this year and will base it on three years of trial analyses showing apparent slowing of disease progression at the highest dose tested. UniQure also highlighted an upcoming FDA discussion in Q2 focused on key elements of a potential Phase 3 trial design, after the agency demanded a new trial before approving AMT-130 in the U.S. The update provides a renewed regulatory route outside the U.S. while the FDA path remains uncertain, keeping attention on how sham-controlled designs and trial redesign requirements shape approvals for rare neurodegenerative gene therapies.
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