FDA feedback confirms tough path forward for uniQure gene therapy in Huntington’s

The final meeting minutes from uniQure’s pre-BLA meeting with the FDA confirm that the agency does not believe that the data submitted from the phase 1/2 studies of AMT-130, uniQure’s investigational gene therapy for Huntington’s disease, provide the primary evidence to support a BLA submission.

The news is consistent with what uniQure shared immediately following the October 29, 2025 meeting, when the company said that it believes that the agency no longer agrees that data from the phase 1/2 studies of AMT-130 in comparison to an external control — as per the prespecified protocols and statistical analysis plans shared with the FDA in advance of the analyses — will be adequate to provide the primary evidence in support of 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.

Now, uniQure says it is carefully evaluating the feedback and plans to urgently request a follow-up meeting with the FDA to take place in the first quarter of 2026.

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.

In September, uniQure shared that AMT-130, met its primary endpoint as well as a key secondary endpoint in a pivotal phase 1/2 trial for Huntington’s disease. In the study, uniQure analyzed clinical outcomes for 29 patients treated with AMT-130 (n=17 high dose; n=12 low dose) of which 12 patients per dose group had attained 36 months of follow up.

Huntington’s disease, a rare, inherited neurodegenerative disorder for which there are currently no disease-modifying therapies available. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain.