Huntington’s Disease Community Advocacy Helps Land Critical FDA Meeting for AMT-130

The Huntington’s disease (HD) community has shown its collective power over the past two months, rallying together to advocate for the continued advancement of AMT-130, uniQure’s investigational gene therapy, in the United States. This sustained advocacy effort appears to have contributed to the scheduling of uniQure’s Type A meeting with the Food and Drug Administration (FDA) to discuss the US regulatory pathway for AMT-130.

Community delivers petitions to the FDA

The HD community’s response to recent regulatory challenges has been nothing short of extraordinary. Two online petitions calling on the FDA to reconsider its position on AMT-130 gathered over 48,000 signatures in a short amount of time, representing an unprecedented show of unity in the HD community. Beyond the digital signatures, community members wrote letters to Congressional representatives and organized meetings with lawmakers to highlight the urgent unmet need for HD treatments.

This advocacy culminated in a powerful moment on January 22, 2026, when HD community representatives made the journey to FDA headquarters in Silver Spring, Maryland, to personally hand-deliver the printed petitions bearing those 48,000+ signatures. Representatives from Help 4 HD International, the Huntington’s Disease Youth Organization (HDYO), the Huntington’s Disease Society of America (HDSA), HD Reach, and the Huntington’s Disease Foundation (HDF) came together for this coordinated action.

This wasn’t just a symbolic gesture, it was a physical manifestation of the HD community’s collective voice. The organizations present represented the full spectrum of the HD community: young people at risk, people living with the disease, family members caring for loved ones, and advocates who have worked for decades toward effective treatments. Their presence at the FDA sent an unmistakable message about the urgency of the need for disease-modifying therapies ahead of uniQure’s scheduled Type A meeting.

The Type A meeting

On January 9, uniQure announced it had scheduled a Type A meeting with the FDA. In a letter to the HD community dated January 20, the company explained what this means.

Type A meetings are reserved for urgent issues. They are designed to help companies resolve critical roadblocks and obtain essential feedback from the FDA on their development programs. These high-priority meetings are typically scheduled within 30 days of the FDA receiving a meeting request. The FDA generally provides official meeting minutes approximately 30 days after the meeting takes place.

Importantly, uniQure has committed to including a community representative at the Type A meeting, ensuring that HD community voice will be heard directly by FDA regulators during these discussions. The company has indicated it will provide a regulatory update once it receives the official meeting minutes from the FDA. So, according to uniQure’s timeline, the community should know more by early March.

The power of community

In their letter, uniQure specifically acknowledged the HD community’s advocacy efforts over the past two months, stating that they have played an important role in raising awareness of the significant unmet need in HD.

This level of coordinated advocacy is unprecedented in the HD community. The 48,000+ petition signatures, Congressional outreach, and organized delivery of petitions to the FDA demonstrate what can happen when people living with the disease, families, young people, and organizations work together toward a common goal.

While we don’t yet know the outcome of uniQure’s meeting with the FDA, the HD community has already accomplished something significant. They’ve made it impossible to ignore the urgent need for effective HD treatments. Every signature, every letter, every conversation with a lawmaker contributed to ensuring that the voices of those affected by HD were heard by decision-makers.

What comes next?

UniQure expects to provide an update by or before early March, after receiving official meeting minutes from the FDA meeting. Until then, the company has encouraged the HD community to continue raising awareness about the urgent unmet need of HD.

The past two months have shown that when the HD community speaks with one voice, people listen. People living with HD, families, at-risk individuals, and advocates have demonstrated their collective strength and determination to fight for access to potentially disease-modifying therapies.

Whatever the ultimate outcome of this meeting, this period of advocacy will be remembered as a defining moment when the HD community stood together and made its needs known, loudly, clearly, and with undeniable impact.

Summary

• Over 48,000 people signed petitions calling on the FDA to reconsider its position on AMT-130
• On January 22, 2026, representatives from five major HD organizations hand-delivered those petitions to FDA headquarters in Silver Spring, Maryland
• UniQure has secured a high-priority Type A meeting with the FDA to discuss the regulatory pathway for AMT-130
• A community representative will participate directly in the FDA meeting