FDA changes stance on uniQure gene therapy data

Following feedback from a pre-BLA meeting with the FDA, uniQure is now questioning the timing for its submission of AMT-130, an investigational gene therapy for Huntington’s disease.

uniQure 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, may be adequate to provide the primary evidence in support of a BLA submission.

According to uniQure, this is a shift from prior communications with the FDA in multiple Type B meetings over the past year.

“This news is unexpected, and we are truly disappointed for people living with HD, who have no disease-modifying treatment options for this devastating disease. We strongly believe that AMT-130 has the potential to bring substantial benefit to patients, and we remain fully committed to working with the FDA to determine the best path forward to rapidly bring AMT-130 to patients and their families in the U.S.,” said Matt Kapusta, uniQure CEO.

The company had originally planned on submitting a BLA for AMT-130 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.

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.