UniQure Secures Type A Meeting with FDA: What This Means for AMT-130

UniQure announced on January 9, 2026 that they have scheduled a Type A meeting with the FDA to discuss the approval pathway for AMT-130 in the United States. Type A meetings are urgent, high-priority discussions reserved for urgent issues, and they happen fast, typically within 30 days. The meeting will focus on determining exactly what data package the FDA would need to support accelerated approval. After two months of uncertainty following November’s regulatory setback, this represents a structured opportunity for both sides to hopefully find a path forward.

A Critical Next Step

Just over two months after the November regulatory setback that left many within the HD community confused and deflated, uniQure announced on January 9 that the FDA has scheduled a Type A meeting to discuss the path forward for AMT-130 in the US. 

While the press release is brief, the meeting type itself tells us something important: this is urgent, and both sides have agreed to come together to try and find a path forward.

What Exactly Is a Type A Meeting?

In the world of FDA interactions, not all meetings are created equal. The FDA offers several types of formal meetings with drug developers, but Type A meetings occupy a special category. They’re reserved specifically for situations where a development program has stalled, like when there’s a clinical hold, major safety concerns, or formal dispute that need to be addressed before a drug can move forward.

Think of it like this: if regular FDA meetings are scheduled check-ins during a road trip, a Type A meeting is pulling over to consult the map when you’ve hit an unexpected detour. It signals both urgency and a genuine attempt to hopefully problem-solve rather than just exchange information.

While the uniQure press release didn’t include a date for this meeting, Type A meetings are scheduled within 30 calendar days of the FDA receiving the request. So we should have more information by early February. 

Type A meetings happen much faster than the 60-75 days typical for other meeting types. This expedited timeline reflects their high-priority nature. The meeting package (all the supporting documents and specific questions) must be submitted at the same time as the meeting request, and the FDA aims to provide preliminary responses just two days before the scheduled meeting.

What Can Companies Gain from Type A Meetings?

Type A meetings serve several purposes. They can help:

• Resolve disputes about trial design requirements or regulatory decisions
• Lift clinical holds by determining what modifications or additional data are needed
• Chart a path forward when a development program has stalled due to regulatory concerns
• Clarify expectations so companies know exactly what the FDA requires

The meetings themselves are typically 60 minutes, focused and structured around a specific agenda agreed upon beforehand. Within 30 days after the meeting, the FDA issues formal meeting minutes summarizing key takeaways, agreed-upon actions, and next steps.

What UniQure Is Seeking

According to the press release, the meeting will focus on “the Biologics License Application (BLA) data package to support accelerated approval of AMT-130.” In other words: what exactly would the FDA need to see in order to grant approval through the accelerated pathway?

This is crucial. Back in November, uniQure learned that their Phase 1/2 data compared to external controls from the Enroll-HD database, which the FDA had previously indicated would be acceptable, was no longer deemed sufficient as the primary evidence for approval. The Type A meeting gives both parties a structured opportunity to discuss what would be sufficient.

CEO Matt Kapusta emphasized the urgency: “The Huntington’s disease community, including patients and clinicians, has emphasized the profound unmet medical need and the importance of timely access to potentially disease-modifying therapies such as AMT-130.”

What Happens Next

UniQure stated they will “provide a regulatory update after receipt of official meeting minutes from the scheduled Type A meeting.” Those minutes should arrive within 30 days of whenever the meeting occurs, which, given the expedited timeline, could be as soon as late January or early February 2026.

The meeting minutes will be critical. They’ll spell out exactly what the FDA believes would constitute adequate evidence for approval, whether that means additional trial data, a different statistical approach, or something else entirely. This clarity is what’s been missing since November.

The Bigger Picture

This announcement represents forward movement, though it’s critical to remember that it doesn’t guarantee a specific outcome. What it does confirm is that both uniQure and the FDA are actively working to find a path forward rather than simply walking away from AMT-130.

The data haven’t changed. AMT-130 still appears to show a slowing of disease progression with a manageable safety profile in the original analysis. What’s being negotiated is the regulatory framework: how to demonstrate that finding in a way that meets the FDA’s standards for approval.

For the HD community, which has signed over 46,000 petition signatures and issued a Statement of Unity from major advocacy organizations, this meeting represents a chance for their voices to be heard, not just in public forums but in the regulatory decisions that ultimately determine drug access.

Meanwhile, uniQure continues to advance regulatory discussions in the EU and UK, providing alternative pathways that could benefit the global HD community regardless of the US outcome.

The coming weeks will be telling. The outcome of the Type A meeting could provide the roadmap needed to bring AMT-130 to patients. Alternatively, it could clarify just how high the remaining hurdles are. Either way, the HD community will finally have more concrete answers than they’ve had since that disappointing November announcement.

Summary

• UniQure announced a Type A meeting has been scheduled with the FDA to discuss AMT-130’s approval pathway
• Type A meetings are high-priority, urgent discussions reserved for urgent issues on stalled development programs
• The meeting will focus on determining what data package could potentially support accelerated approval
• Type A meetings typically occur within 30 days and are designed to resolve critical regulatory roadblocks, so we should know more by early February
• UniQure will provide an update after receiving official meeting minutes