uniQure to seek UK nod for Huntington’s gene therapy, following FDA setback

uniQure, following a pre-submission meeting with the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), says it will submit a marketing authorization application for its gene therapy, AMT-130, for the treatment of Huntington’s disease in the third quarter of 2026.

Following discussions with the MHRA, uniQure expects to submit its application based on the three-year analysis from the ongoing U.S. and European phase ½ clinical trials. At year three, these data showed a 75% slowing of disease progression at the high dose as measured by the composite Unified Huntington’s Disease Rating Scale compared to a propensity score-matched external control, with a manageable safety profile.

Last month, uniQure announced that it had received final Type A meeting minutes from the U.S. FDA regarding 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 meeting strongly recommended that uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study — feedback that uniQure said was a shift from prior communications with the FDA in multiple Type B meetings over the past year.

Now, uniQure says it has been granted a Type B meeting with the FDA in the second quarter of 2026. The company expects to discuss key elements of a potential phase 3 trial design.

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.