uniQure eyes 2026 for AMT-130 approval application in US

Company says it's aligned with FDA on analysis, manufacturing requirements

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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uniQure plans to submit a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) early next year seeking approval of its gene therapy candidate AMT-130 for Huntington’s disease.

Top-line, three-year data from the ongoing clinical trials that will support that application are due in the next few months, and uniQure plans to hold a pre-submission meeting with the agency by year’s end.

The company said it has aligned with the FDA on key aspects of the application, including its statistical analysis plans for the trials as well as chemistry, manufacturing, and controls (CMC) requirements.

“We are very pleased with our continued, productive engagement with the FDA and the progress we’ve made toward a planned BLA submission for AMT-130 in the first quarter of 2026,” Walid Abi-Saab, MD, uniQure’s chief medical officer, said in a company press release.

The BLA will be filed through the FDA’s accelerated approval pathway, which enables regulators to give conditional marketing authorization to a treatment that could fill an unmet need for a serious disease based on early trial data suggesting it will be of clinical benefit to patients. Conversion to a full approval requires additional trial data to confirm such benefits.

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Company will seek priority review

uniQure will seek priority review designation for the application, which would shorten the review time from a standard 10 months to six months.

“We are pursuing an accelerated approval pathway supported by multiple years of clinical data — a rigorous and differentiated approach that reflects the urgent need in Huntington’s disease and our commitment to delivering the first disease-modifying treatment for people affected by this devastating disease,” Abi-Saab said.

The company’s ability to seek AMT-130’s approval through these pathways is facilitated by the therapy’s fast track and breakthrough therapy designations, which give developers more frequent interactions with the FDA and can make a therapy eligible for accelerated and priority review if certain criteria are met.

AMT-130 also has orphan drug and regenerative medicine advanced therapy designations in the U.S., which offer other incentives to speed its clinical development.

Huntington’s is caused by mutations in the HTT gene that result in the production of an abnormal form of the huntingtin protein that’s toxic to nerve cells.

A one-time gene therapy, AMT-130 is designed to deliver a small piece of genetic material called a microRNA that aims to silence the HTT gene in order to lower mutant huntingtin production. This is expected to ease Huntington’s symptoms and slow overall disease progression.

The treatment is packaged in a viral carrier that helps it get into cells, and is administered directly into the brain via a surgical procedure.

AMT-130 is being tested at two dose levels in a pair of Phase 1/2 trials, one that’s being conducted in the U.S. (NCT04120493) and another in Europe (NCT05243017). Interim data from these studies have shown that AMT-130 could slow disease progression and lower mutant huntingtin levels in adults with early-stage Huntington’s.

The FDA agreed late last year that data from these studies could support a BLA for AMT-130’s accelerated approval.

uniQure said it has since held two meetings with the agency. In one, the FDA agreed on the statistical analysis plans, including an outcome measure that may support an accelerated approval.

The trials’ main goal will be to compare three-year changes in scores on the composite Unified Huntington’s Disease Rating Scale, a standard measure of disease progression, between people given the high AMT-130 dose and an external control group.

This control group will consist of untreated Huntington’s patients participating in Enroll-HD (NCT01574053), the world’s largest observational study on Huntington’s. A statistical method called propensity score weighting will be used to adjust for potential differences between the AMT-130 and control groups and better assess the treatment’s effect.

uniQure will submit to the FDA an updated statistical analysis plan that aligns with these discussions in the coming months.

In another meeting, the company aligned with the agency on various CMC requirements related to AMT-130’s manufacturing. The regulators agreed that the manufacturing process is feasible based on prior experience with the approved hemophilia gene therapy Hemgenix, which uniQure originally developed.

The company will initiate a quality control test run of the manufacturing process later this year.

“We are grateful to the FDA for their continued engagement and look forward to sharing three-year top-line data” between July and September, Abi-Saab said.