Petition to the U.S. Food and Drug Administration

Approve AMT-130 for Huntington’s Disease: Give Families Hope Now

To: The U.S. Food and Drug Administration (FDA)
From: The Huntington’s Disease (HD) Community: patients, caregivers, and loved ones united for hope

Why This Matters

We, the undersigned, write with urgency on behalf of the HD community. In a historic milestone, AMT-130 by uniQure has become the first treatment to show potential in slowing the progression of this devastating genetic disease. This is more than a scientific milestone, it is a lifeline for thousands of families who have waited generations for a glimmer of hope.
 
Facts About Huntington’s Disease

Huntington’s Disease (HD) is a rare, inherited brain disorder that destroys nerve cells over time. It robs people of their ability to move, think, speak, and care for themselves—striking often during their prime working years. 

• Every child of a parent with HD faces a 50% chance of inheriting the faulty gene that causes the disease.
• Today, about 41,000 Americans live with HD, and more than 200,000 are at risk.
• HD is often described as having ALS, Parkinson’s, and Alzheimer’s combined.
• The toll is crushing: spouses become full-time caregivers, children watch parents decline, and families lose their stability, savings, and hope.
• Life expectancy drops to just 15–18 years after symptoms appear.
• Tragically, people with HD are 9–10 times more likely to die by suicide than their peers, making suicide one of the top causes of death in HD.
  These are not statistics. They are lives waiting for help. Every delay deepens the suffering.

The Promise of AMT-130

• AMT-130 is a one-time gene therapy designed to target the root cause of Huntington’s disease, the toxic mutant huntingtin (mHTT) protein. 
• Early data from uniQure’s Phase I/II study suggest that AMT-130 may slow disease progression by up to 75% in treated patients compared to well-matched controls, with a strong safety profile.
• For HD families, this could mean more time, more independence, and more dignity. It could mean parents witnessing their children grow up, and children free from the fear of their genetic destiny.

Regulatory Progress and Setback

AMT-130 has already earned multiple FDA designations recognizing its extraordinary promise:

• Orphan Drug Designation
• Regenerative Medicine Advanced Therapy (RMAT) Designation
• Fast Track Designation
• Breakthrough Therapy Designation
• In December 2024, the FDA confirmed that AMT-130 was eligible for the Accelerated Approval pathway, created specifically for serious, life-threatening diseases with no approved treatments.

But in October 2025, the FDA reversed that position, stating that the current Phase I/II data were “not adequate” to support a Biologics License Application (BLA) at this stage.
That decision halted progress on what could be the first treatment to slow Huntington’s disease.

Why Immediate Action Is Essential

• Families affected by HD do not have the luxury of time.
• Every month of delay means more loss of function, independence, and life.
• Other rare diseases have received accelerated approval based on external-control data, even those affecting far fewer people.
• With over 30,000 participants in the Enroll-HD study, the HD community already provides one of the largest and most reliable external datasets in rare disease research.
• We are not asking for shortcuts, we are asking for fairness, consistency, and urgency.
• If other rare and fatal diseases can access accelerated approval, then Huntington’s families deserve the same compassion and opportunity.

Our Call to the FDA

We, the undersigned, respectfully urge the FDA to:

1. Allow uniQure to submit a Biologics License Application (BLA) for AMT-130 under the Accelerated Approval pathway, using existing Phase I/II data and validated external controls.
2. Recognize the severe unmet medical need and the strong preliminary evidence already shown.
3. Act with the urgency and flexibility given to other rare and fatal disease communities.
4. Empower HD patients and families with the right to choose this therapy when no other options exist.
    

Our Plea: Turn Heartbreak Into Hope

• We are mothers and fathers, sons and daughters, brothers and sisters, husbands and wives, friends and caregivers—all united by love and by loss.
• AMT-130 could change everything.
• It could give families more time.
• It could give children a future free from fear.
• Waiting is not caution. Waiting is loss, and loss we can prevent.

Please, FDA, ACT NOW.
Approve AMT-130 under the Accelerated Approval pathway.
Give HD families the chance to live the lives they deserve.
 

Signed,
Members of the Huntington’s Disease Community
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