We received an update on June 2, 2025 from uniQure about their recent discussions with the US Food and Drug Administration (FDA) regarding the development of AMT-130 – a treatment they’re testing for Huntington’s disease (HD). uniQure announced that they remain aligned with the FDA and have received guidance on next steps, including plans for manufacturing, statistics, and a comparison control group. So what exactly did we learn from this recent update? Let’s get into it!
Laying the Tracks
We’ve been following the development of uniQure’s HTT-lowering drug, AMT-130, from its genesis in the lab, to animal studies just a few years ago, and now through to their ongoing Phase 1/2 clinical trials.
We’ve also written about it quite a bit. To save our regular readers from repetition, we’ll link any HDBuzz newbies to previous news we’ve covered:
- Promising results from animal models, covered in April, 2021
- The announcement of the clinical trial, covered in July, 2021
- 12 month safety data, covered in July, 2022
- A short pause in the trial, covered in August, 2022
- The trial resumes, covered in November, 2022
- A promising safety profile, covered in December, 2023
- AMT-130’s potential to slow clinical progression, covered in July, 2024
- Previous alignment with the FDA, covered in December, 2024

FDA Meetings
On June 2nd, CEO Matt Kapusta shared that uniQure had two milestone meetings, called Type B meetings, with the FDA. These are formal meetings between the regulatory agency and drug companies to discuss development and advancement of a medicine. Any face-to-face meeting with the FDA is a good opportunity for a drug company, because it helps to ensure that they’re moving in a direction that the agency supports.
During these meetings, uniQure aligned with the FDA on a plan for submitting a Biologics Licensing Application (BLA). This is the formal application a drug company puts together to request permission to market a biologic, a type of therapy derived from living organisms, such as proteins, cells, or genetic material. Unlike traditional small-molecule drugs, biologics are typically larger and more complex, and include treatments like antibodies and gene therapies. UniQure’s Chief Medical Officer, Dr. Walid Abi-Saab, shared that the FDA agreed that data collected from their ongoing trials could be used to support a future BLA. In other words, uniQure may not need to run another trial before seeking accelerated approval for AMT-130.
The most exciting part? UniQure shared that their hope is to submit this BLA for accelerated approval during the first quarter of next year – as soon as the end of March, 2026. BUT, that doesn’t mean this is a done deal. The upcoming data, anticipated by the end of September 2025, still has to show that the drug is having the expected effect, or else the FDA won’t approve the BLA.
For serious conditions like HD, which carry a tremendous burden with limited treatment options, regulators may greenlight earlier access to drugs that show potential.
Approval Paths
As HD drugs advance through the clinic, we’ve been hearing hopeful news about “accelerated approval.” But what exactly does that mean, and how does it differ from traditional approval?
First, the easy one – traditional approval. This means that a drug has undergone rigorous testing, is safe and well tolerated, and has improved symptoms or delayed worsening of a disease. Drugs that gain traditional approval have lots and lots of data to support their use, and they are very unlikely to be pulled from the market.
With accelerated approval, a regulatory agency allows a drug to be made available to the general population while the drug is still being tested. There isn’t enough data yet to conclusively say that it’s having the desired effect. However, for serious conditions like HD, which carry a tremendous burden with limited treatment options, regulators may greenlight earlier access to drugs that show potential.
The idea is that the accelerated approval path could help people in need access drugs sooner, in hopes that they will work. But drugs made available through accelerated approval could still be pulled from the market if additional testing suggests that they don’t actually benefit patients. While no one wants to take drugs that don’t work, many people accept the risk of taking drugs that might work, which is why uniQure is exploring the accelerated approval path.

Regulatory milestones
To make sure that uniQure and the FDA are aligned on steps towards a speedy (possible) approval of AMT-130, they talked about a few key points:
Manufacturing
Thinking ahead, the FDA wants to know if uniQure is capable of manufacturing AMT-130 at scale for people with HD, should the data continue to look good. UniQure shared that the FDA agrees they have the manufacturing capabilities and has endorsed their production platform. This streamlines uniQure’s path toward commercial readiness and a future BLA submission.
Statistical Plan
During the meeting, the FDA and uniQure also talked about the type of data they want to see and how those data should be analyzed. Previously, the FDA agreed that positive data for the composite Unified Huntington’s Disease Rating Scale, or cUHDRS, could be used to decide whether the drug is working. They also discussed a biomarker called NfL, which increases as HD progresses. They agreed that if AMT-130 reduces NfL, it could be taken as a positive sign that the treatment is working.
This is exciting news because it sets the bar for what needs to be achieved to have the FDA approve a drug for HD. UniQure’s announcement confirmed that the FDA is still in support of using these metrics to help decide whether an HD drug is working.
They’ve also agreed on exactly what types of statistical tests should be used to analyze the data. It’s great to know that the FDA and uniQure are aligned on the nitty gritty, but we’ll spare you those mathematical details here!
Control Group
Another important point the agency agreed on was which dataset uniQure should use to compare their trial results. While the first cohort included people who received a mock surgery, the ongoing AMT-130 studies don’t include a traditional placebo group of participants who did not receive the drug. Since this a long-term study being conducted over several years, it was instead designed to compare people receiving AMT-130 to participants in a natural history study.
In a natural history study, no drug is given. It’s designed to follow people who have the gene for HD and to observe them as they naturally live and age to see how HD progresses. These types of studies give us a wealth of information.
UniQure were previously comparing AMT-130 recipients to those who participated in the TRACK-HD, TrackOn-HD, and PREDICT-HD studies. They’re now also incorporating data from Enroll-HD – the world’s largest observational study for HD families. The Enroll-HD dataset will be used for the primary analysis, and data from TRACK-HD and TrackOn-HD will be used for additional analysis.
This change was made because the Enroll-HD dataset is significantly larger, at almost 33,000 people enrolled (wow!), many of whom fall into the inclusion criteria for the AMT-130 trials. This gives a better comparator when looking at the data and increases the chances of seeing a stronger effect of AMT-130.
They’ll be presenting this data publicly during the third quarter, so we should have a much better idea of AMT-130’s 3-year effectiveness by the end of September, 2025. After that, they plan to meet again with the FDA in the fourth quarter of 2025, to prepare for submitting their BLA in the first quarter of 2026.
The Track Ahead
On the June 2nd investor call, uniQure gave us a date for when they would stop collecting data to send over to the FDA – June 30. That cutoff will include data from people who were given AMT-130 3 years ago, so it’s quite a long-term follow up.
They also shared that they’ll be presenting this data publicly during the third quarter, so we should have a much better idea of AMT-130’s 3-year effectiveness by the end of September, 2025. After that, they plan to meet again with the FDA in the fourth quarter of 2025, to prepare for submitting their BLA in the first quarter of 2026. At that meeting, uniQure will request that the FDA make their review a priority.
To continue laying the track to reach an HD gene therapy, the data must continue to be positive. In the meantime, we’ll be standing on the platform, waiting to let you know when the whistle blows!
TL;DR Summary
- A recent meeting between the FDA and uniQure continues to show alignment
- cUHDRS and NfL lowering continue to be agreed upon metrics for success
- UniQure got a green light from the FDA on their manufacturing platform
- Statistical plans for data analysis were discussed and agreed upon
- Enroll-HD will be used as the natural history control group for primary analysis
- Next data update – Q3 2025, which will include 3 year follow up data
- Q4 2025 meeting with the FDA to discuss accelerated approval
- Plan to submit Biologics Licensing Application for accelerated approval in Q1 2026
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