The FDA is delaying a lifeline for Huntington's patients by hesitating on accelerated approval for uniQure’s promising AMT-130 gene therapy. This treatment could slow the fatal progression of the disease. We urge the FDA to honor its prior agreements and expedite review. For the Huntington's community, who live without hope and are running out of time, every minute counts.

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Background: A Glimmer of Hope

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Huntington’s Disease (HD) is a fatal, genetic neurological illness that causes the progressive breakdown of nerve cells in the brain. It has no cure and robs individuals of their physical and mental abilities.

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For years, the HD community has had no viable treatment options. That is, until now. The gene therapy AMT-130, developed by uniQure, has shown significant promise in slowing disease progression. The FDA itself has acknowledged this potential, granting AMT-130 both Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations. These are reserved for drugs that may demonstrate substantial improvement over available therapy for serious conditions.

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The FDA and uniQure had previously agreed on an accelerated approval pathway—a process designed specifically for devastating diseases like HD. This pathway allows for earlier approval based on data that reasonably predicts a clinical benefit, with the understanding that more data will be collected after approval.

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The Present Crisis: A Promise in Peril

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The FDA is now wavering on its commitment. It is reconsidering the use of a "natural history" group as a control for the study—a scientifically valid and widely accepted method for rare diseases where using a placebo is unethical. This method has strong precedent and is supported by massive studies like Enroll-HD, which has collected data from over 30,000 people to map the natural progression of this disease.

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By casting doubt on its prior agreements, the FDA is single-handedly jeopardizing the most promising path forward for a community that has no other options. This isn't just a bureaucratic delay; it is a direct threat to the lives and hopes of thousands of patients and families.

 

Our Requests& Recommendations

 

We are petitioning for immediate action. The time for waiting is over.

Recommendations for the FDA:

• Honor Your Agreement: Fully uphold the previously agreed-upon accelerated approval pathway for AMT-130, including the use of external control data from natural history studies.

• Recognize the Urgency: Acknowledge the dire, unmet medical need in the HD community and act with the expediency that Breakthrough Therapy and RMAT designations demand.

• Expedite Review: Make the review and approval of AMT-130 a top priority to get this potentially life-altering therapy to patients without further delay.

 

Call to Action for the HD Community:

• Sign and Share: Your name on this petition adds power to our collective voice. Share it with your family, friends, and support networks.

• Contact Your Representatives: Tell your story and demand they hold the FDA accountable.

• Amplify Your Voice: Use social media to share why this therapy is so critical.

 

Call to Action for the General Public:

• Show Your Support: Sign this petition to stand in solidarity with the tens of thousands of families affected by Huntington's Disease.

• Educate Yourself: Learn about the devastating impact of HD and understand why accelerated access to treatment is so critical.

• Spread the Word: Share this cause. You have the power to help us move mountains.

 

We cannot allow procedural hesitation to become a death sentence. Sign now.