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 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.’”
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