The gene therapy developer, uniQure NV, has asked for a second meeting with the US Food and Drug Administration to discuss the agency’s recommendation for further clinical development of its candidate therapy for Huntington’s disease AMT-130. The gene therapy is intended as a one-time treatment for Huntington’s, a rare neurodegenerative disorder. uniQure produced data from a Phase 1/2 trial in September 2025 which was intended as a pivotal study leading to a regulatory submission.
However in January the FDA requested further evidence. This would need to come from a Phase 3 sham-surgery controlled study, according to uniCure. A sham study is an inactive, placebo-like study designed to mimic a medical procedure in order to eliminate bias.
AMT-130 is administered directly to the brain. The therapy uses an adeno-associated virus to deliver microRNA directly to the stratum in order to induce the silencing of a mutant huntingtin gene and subsequently toxic proteins. When functioning normally, the huntingtin protein provides essential scaffolding for the brain. In the company’s Phase 1/2 study, AMT-130 showed that in 12 patients at the highest dose, the therapy delivered a 75% slowing of disease progression compared with an external control group.
“While we did not reach alignment on a submission pathway based on the Phase 1/2 data, we believe the totality and durability of our data warrant continued substantive dialogue regarding how the FDA’s stated commitment to regulatory flexibility may be appropriately applied in this setting,” said Matt Kapusta, the company’s chief executive, in a prepared statement.
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