UniQure said it plans to submit a marketing application for its Huntington’s gene therapy AMT-130 to UK regulators in the third quarter, after a positive meeting with drug regulators there. The submission would be supported by a three-year analysis from ongoing U.S. and Europe trials, the company said. The update comes amid an ongoing U.S. review pathway challenge, where UniQure previously said FDA demanded a new trial before approval after requesting a new sham-controlled design. UniQure said it will also meet FDA in the second quarter to discuss elements of a potential Phase 3 trial. For Huntington’s, the UK filing represents a critical fork in the road for patients awaiting disease-slowing therapies, while highlighting the regulatory divergence gene-therapy developers face across geographies.
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