FDA rejects uniQure data package for Huntington’s gene therapy

uniQure announced that it has received final Type A meeting minutes from the U.S. FDA regarding its investigational Huntington’s disease gene therapy, AMT-130, with the agency stating that data from the phase 1/2 studies are not sufficient to support a marketing application.

The minutes from the January 30, 2026 meeting strongly recommended that uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study.

“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, uniQure CEO.

The Type A meeting feedback aligns with updates uniQure shared in December, when the company announced that final meeting minutes from its pre-BLA meeting with the FDA indicated the agency did not consider data from the phase 1/2 AMT-130 studies sufficient as the primary evidence to support a BLA submission. According to uniQure, this was a shift from prior communications with the FDA in multiple Type B meetings over the past year when the company had aligned with agency on a plan to compare AMT-130 to a natural history external control.

uniQure stock tumbled 32.8% with the recent news. uniQure says it intends to continue engaging with the FDA regarding phase 3 development considerations and plans to request a Type B meeting in the second quarter of 2026 to further discuss potential study design approaches.

AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging the company’s proprietary miQURE silencing technology. The therapeutic goal is to inhibit the production of the mutant protein. AMT-130 has been granted breakthrough therapy designation and regenerative medicine advanced therapy (RMAT) designation from the FDA.