‘Ready to fight the fight’: Huntington’s disease community delivers petitions to FDA seeking approval of uniQure gene therapy

  

On January 22 Huntington’s disease advocacy organizations delivered two petitions to the U.S. Food and Drug Administration (FDA), demanding that the agency reverse its rollback on uniQure’s application for its HD gene therapy.

 

In September, uniQure announced that its drug, AMT-130, had slowed the progression of HD by 75 percent over three years – a historic first. However, reflecting what critics saw as growing dysfunction under the Trump administration, the FDA abruptly switched gears regarding its agreed-to plan for considering AMT-130.

 

HD advocates quickly organized two online petitions. By January 17, they had garnered more than 48,000 signatures.

 

Advocates from HD Reach, Help4HD International, the Huntington's Disease Foundation, the Huntington’s Disease Society of America (HDSA), and the Huntington’s Disease Youth Organization (HDYO) jointly delivered the printed petitions to FDA headquarters in Silver Spring, MD.

 

“We can’t delay any longer,” Lauren Holder, a Help4HD advocate and (like me) an HD gene carrier desperate for a therapy like AMT-130, said in a Facebook video. “It’s important that they hear us.”

 

Surviving HD

 

The 48,000-plus signatures “is not something that the HD community has ever done before,” Holder said. In addition, the “outcry” by advocates to Congress was highly effective, she added.

 

The HD community’s “wonderful job” in speaking up is “important for clinical trials in the future” of gene therapies and other gene-modifying drugs, she said.

 

Holder said that she often hears from HD-affected individuals that “‘I don’t want to be dying from HD; I want to be living with HD.’ These gene therapies and gene-modifying drugs are what get us to that point.”

 

Ramping up the fight

 

In a final, intense push for signatures, Holder on her January 16 podcast interviewed Jeremy Renz, of St. Augustine, FL. His wife tested positive for the HD gene in 2024 and his mother-in-law is in the late stages of the disease. Their family has been affected for many generations.

 

In 2024, Renz was a panelist at the FDA headquarters for a listening session with the HD community, similar to the first such meeting organized by HDSA in 2015, which I attended.

 

Whereas Help4HD and other organizations advocated for one petition, Renz organized another for those families not necessarily linked to formal HD groups.

 

“There’s more young people that are testing,” Renz told Holder. “There’s more young people who are at risk that are getting ramped up and ready to fight the fight.”

 

HD family member Jeremy Renz with petitions advocating for the approval of uniQure's AMT-130 at FDA headquarters, Silver Springs, MD, January 22, 2026 (Facebook photo)

 

A crucial meeting

 

On January 9, uniQure announced that it had scheduled a so-called Type A (high-priority) meeting with the FDA, to take place within 30 days, “to support accelerated approval of AMT-130.”

 

On January 20, Daniel Leonard, uniQure’s executive director of global patient advocacy, distributed a letter to the HD community explaining the purpose of the Type A meeting.

 

“Type A meetings are intended to address urgent issues and help a sponsor, such as uniQure, resolve developmental roadblocks and obtain critical feedback from the FDA,” Leonard wrote. “The extraordinary efforts of the HD community over the past two months have played an important role in raising awareness of the significant unmet need in HD.”

 

Crucially, the Type A meeting will include a representative of the HD community, as of now unnamed.

 

Leonard stated that uniQure will provide an update after receiving the official meeting minutes from the FDA.

 

The HD community has made a difference

 

In their podcast conversation, Holder and Renz noted that the FDA’s backtrack on the AMT-130 plans was “infuriating.”

 

“It’s really, really frustrating for a community that deserves a lot,” Renz said. “We deserve an answer. We deserve treatment. And the time is now.”

 

For HD gene carriers like herself, Holder said a delay in receiving a drug such as AMT-130 reduces the “window” of opportunity for treatment.

 

Holder and Renz concluded by saluting how the HD community had united to work hard on AMT-130 advocacy.

 

“We are definitely stronger together,” Holder said. “We are living history right now. This is something that everybody is going to look back on and say: ‘The community made a difference here, and they pushed through at the right point.’”

uniQure schedules critical FDA meeting to revisit request for approval of Huntington’s disease drug

 

uniQure announced on January 9 that it has scheduled a high-priority meeting with the U.S. Food and Drug Administration (FDA) to revisit its application to seek approval of its gene therapy drug for Huntington’s disease, after the agency had rolled back its permission.

 

As recounted here, uniQure and others in the frustrated biotech sector believe that the FDA has become dysfunctional under the Trump administration (click here and here for details).

 

In September, uniQure announced that its drug, AMT-130, had slowed the progression of HD by 75 percent over three years – a historic first. However, in November uniQure announced that the FDA had abruptly switched gears regarding its agreed-to plan for considering AMT-130.

 

In its January 9 press release, uniQure stated that it had scheduled a so-called Type A meeting with the FDA “to support accelerated approval of AMT-130.” It did not disclose the date of the meeting.

 

“We look forward to a constructive discussion with the FDA as we work toward a timely resolution regarding an accelerated approval pathway for AMT-130,” stated uniQure CEO Matt Kapusta. “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. We remain deeply committed to patient access while continuing to collaborate closely with the FDA.”

 

Seeking to resolve the dispute over AMT-130

 

As noted by the scientist-written website HDBuzz, Type A FDA 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,” HDBuzz explained.

 

Type A meetings are scheduled within 30 days of a request, HDBuzz stated, adding that a Type A meeting can help to “resolve disputes” about clinical trial design or regulatory decisions, “chart a path forward,” and “clarify expectations” so that firms know precisely what the FDA requires. (Click here for the FDA’s official explanation of its meeting types.)

 

Based on this timeline, news from the meeting could be reported in late January or February. The uniQure release stated that the company would provide an update after receiving official minutes from the FDA.

 

Pressing our leaders

 

As of January 12, nearly 47,000 individuals had signed the two petitions from the HD community urging the FDA to expedite approval of AMT-130.

 

The Huntington’s Disease Society ofAmerica also has urged advocates to contact U.S. representatives and senators to “back fair review of AMT-130” by the FDA (click here to write your representatives).

 

In addition to signing one of the petitions and writing to Congress, I will call my Senators and Representative to explain the importance of this cause for the HD community and beyond.

 

I urge everyone to press our political leaders on AMT-130.

 

As I wrote last month, “the FDA should be on the right side of history in this urgent fight to end devastating diseases.”

 

Huntington's Disease Society of America alert: urge Congress to back fair review of AMT-130 (screenshot by Gene Veritas, aka Kenneth P. Serbin)