On December 4, 2025, uniQure announced they have received the final meeting minutes from their October 29 pre-Biologics License Application (BLA) meeting with the FDA regarding AMT-130. The minutes confirm what was reported in early November: the FDA currently believes the Phase I/II data are unlikely to provide the primary evidence needed to support a BLA submission at this time.

While the most recent press release doesn’t provide new information beyond what we already knew, it does represent an important procedural step. uniQure now has the official written record from the FDA meeting, which will be crucial as they work to chart the path forward.

What Happens Next

UniQure has stated they are carefully evaluating the FDA’s feedback and plan to urgently request a follow-up meeting with the agency in the first quarter of 2026. This meeting will be critical for understanding exactly what additional evidence or analyses the FDA requires.

Matt Kapusta, uniQure’s CEO, emphasized the company’s commitment: “We are committed to collaborating with the FDA to advance AMT-130 to patients and their families as rapidly as possible. The support we have seen these last weeks from the Huntington’s disease community, including patients, families, caregivers, clinicians and advocates reinforces the urgency of the unmet need in Huntington’s disease.”

UniQure has stated they are carefully evaluating the FDA’s feedback and plan to urgently request a follow-up meeting with the agency in the first quarter of 2026.

The Community Responds

The HD community has not remained silent through the whiplash of the FDA’s about-face from just 5 months prior, when they stated that data from the ongoing trials would be sufficient to support accelerated approval. 

In response to this challenging moment, major HD advocacy organizations have come together to issue a Statement of Unity (see More Info section below). Help4HD, HDReach, Huntington’s Disease Society of America, Huntington’s Disease Foundation, and the Huntington’s Disease Youth Organization have pledged to work in partnership to champion the voices of those impacted by HD, particularly when communicating with regulatory bodies like the FDA. This collaboration will focus on broader, shared priorities that affect the many therapeutic approaches currently being developed for HD, ensuring that the lived experiences of families are heard and represented in regulatory discussions.

Multiple Change.org petitions urging the FDA to uphold its accelerated approval pathway for AMT-130 have gathered significant momentum, with tens of thousands of signatures from families, caregivers, and advocates gathered in a matter of weeks – 41,805 at the writing of this article. These petitions and their strong support highlight the urgent unmet need in HD and the devastating impact the regulatory uncertainty has on families who finally saw hope on the horizon.

If you’ve not yet signed these petitions and would like to add your voice, you can find them here:

• Bring Hope to Huntington’s Disease Families: Urge the FDA to Uphold Accelerated Approval
• Accelerate Breakthrough Drug Approval for Huntington’s Disease – uniQure AMT-130

The Data Remain Strong

It’s crucial to remember that nothing about the FDA’s position changes the clinical data themselves. AMT-130 still appears to show a 75% slowing of disease progression compared to matched controls, the strongest evidence we’ve ever seen for a disease-modifying therapy in HD. The treatment continues to show a manageable safety profile with no new drug-related serious adverse events reported since December 2022.

Looking Beyond U.S. Borders

While the U.S. regulatory path has hit an unexpected obstacle, AMT-130 continues to advance in other parts of the world. UniQure has stated they are progressing discussions with regulatory agencies in the European Union and United Kingdom. 

If AMT-130 receives approval in these regions, it would benefit people with HD globally, as clinical data from any regulatory jurisdiction strengthens the evidence base and could ultimately support approval elsewhere.

While the timeline is less certain than we hoped, the goal of bringing an effective disease-modifying therapy to the HD community remains firmly in sight, and it’s clear that the HD community is ready to stand up and fight to make that happen sooner than later.

Why This Matters

The regulatory back-and-forth has been emotionally exhausting for the HD community, and we share in your frustration. However, this is not the end of the road for AMT-130. As we’ve said before, this represents a “save point” in HD drug development, a place where solid evidence exists that we can build upon, learn from, and advance forward. The data showing that HTT-lowering seems to slow disease progression remains a landmark achievement, regardless of regulatory timelines.

UniQure has voiced its commitment to seeing AMT-130 through this process. The company has the official meeting minutes, they’re preparing for urgent follow-up discussions, and they’re exploring multiple regulatory pathways. While the timeline is less certain than we hoped, the goal of bringing an effective disease-modifying therapy to the HD community remains firmly in sight, and it’s clear that the HD community is ready to stand up and fight to make that happen sooner than later.

We’ll continue to follow developments closely and keep the HD community informed as new information emerges.

Summary

• UniQure received official FDA meeting minutes from October 29 meeting, confirming Phase I/II data currently unlikely to support BLA submission
• Company plans to request urgent follow-up meeting with FDA in Q1 2026 to determine path forward
• Major HD advocacy organizations issued Statement of Unity, proposing to coordinate efforts with regulatory agencies
• Community petitions gathered 41,805+ signatures urging FDA to uphold accelerated approval pathway
• Clinical data remain unchanged: AMT-130 appears to slow disease progression by 75% with strong safety profile
• UniQure advancing regulatory discussions in EU and UK as parallel pathways
• This represents a regulatory delay, not the end of AMT-130’s development

More Info