FDA grants priority review to uniQure’s Huntington’s therapy

The FDA has accepted uniQure’s application for ifezuntirgene inilparvovec, also known as AMT-130, under priority review for Huntington’s disease, according to Fierce Biotech. uniQure said the agency also tentatively plans to discuss the application at an advisory committee meeting.

Fierce Biotech reported that uniQure said its submission was based on an analysis from its phase 1/2 study. The company described approval as a defining milestone.

Application centers on AMT-130

According to Fierce Biotech, AMT-130 incorporates a miRNA designed to silence the huntingtin gene. The therapy is delivered directly into the brain through small holes in the skull.

Fierce Biotech reported that uniQure previously said patients receiving the higher dose in its phase 1/2 study showed statistically significant slowing of disease progression. The publisher also reported that a later analysis showed slowing on the composite Unified Huntington’s Disease Rating Scale but fell short of statistical significance.

Confirmatory trial planning is underway

uniQure said it aligned with the FDA on key elements of a confirmatory trial, Fierce Biotech reported. The study will enroll patients randomized to receive either the company’s gene therapy or standard of care.

According to Fierce Biotech, the primary endpoint will measure total functional capacity. uniQure said it plans to begin patient screening so the study is underway by the time of an approval decision.

Regulatory path has changed over time

Fierce Biotech reported that AMT-130 faced a dosing pause after safety signals in an open-label, European phase 1/2 trial. The pause was later lifted.

The publisher also reported that the FDA later no longer agreed that the phase 1/2 data were sufficient to support an approval filing, before giving uniQure the go-ahead to file for accelerated approval. The priority-review acceptance is the latest regulatory event for the program.