As uniQure seeks to overcome a decision by the U.S. Food and Drug Administration (FDA) to roll back its consultations regarding the firm’s promising gene therapy for Huntington’s disease, the HD community has begun to rally by organizing two petitions asking the agency to support the remedy.
uniQure and others in the biotech sector believe that the FDA has become dysfunctional under the Trump administration.
In September uniQure announced that its drug, AMT-130, had slowed the progression of HD by 75 percent over three years.
On November 3, uniQure announced that, after a recent meeting with the FDA, it believes that the “FDA currently no longer agrees” that data from its clinical trial of AMT-130 "may be adequate to provide the primary evidence in support of” an application for approval.
The company’s plan to seek approval of AMT-130 in early 2026 and launch it into the market later that year may no longer be possible.
Although the FDA claims it will “unleash gene therapies,” the documented collapse of the agency under the Trump administration, a more conservative view of gene therapies, and “mistrust and paranoia” in the division in charge of those therapies set the stage for backtracking on AMT-130 (click here to read more).
On November 13, the key biotech site STAT reported on a private dinner held by uniQure CEO Matt Kapusta with investors on November 11. According to the report, uniQure hopes to find a way forward for AMT-130.
“We remain fully committed to people living with HD, who have no disease-modifying treatment options,” Tom Malone, uniQure’s senior director of communications, wrote me via e-mail on November 14. “We are wholly focused on working with the FDA to determine the best path forward to rapidly bring AMT-130 to patients and their families in the U.S.”
The company is withholding further public comment until it receives official final minutes of its most recent meeting with the FDA.
Two groups of HD advocates have launched petitions to the FDA to support the original uniQure timeline for AMT-130. They are discussed below.
The online petition to the FDA titled "Bring Hope to Huntington's Disease Families," on November 15, 2025 (screenshot by Gene Veritas, aka Kenneth P. Serbin)
Deep frustration with the FDA
The STAT report noted that Kapusta “doesn’t like all the drama” inside the FDA surrounding its upending of uniQure’s plans. They had involved extensive consultations with the FDA in 2024.
Aiming to stabilize the agency, the FDA has named Richard Pazdur, M.D., a leading cancer specialist and 26-year veteran of the entity, to run its center for regulating and approving new drugs.
Dr. Pazdur’s appointment is a sign the Trump administration is seriously addressing the “FDA dysfunction,” Kapusta said at the investor dinner, as reported by STAT.
According to the STAT report, Kapusta’s remarks at the dinner “reflected biotech’s frustration with volatility” at the FDA. uniQure was disturbed by the fact that the FDA’s new message on AMT-130 was “delivered by lower-level staffers” and not senior decision-makers, STAT reported.
The firm “feels like it was screwed over by the FDA, and rightfully so,” one investor told the STAT reporter.
Meanwhile, the FDA and some scientists have moved ahead with researching and approving the world’s first personalized gene-editing treatments for individuals with rare genetic conditions. It is unclear how much this might benefit rare disease communities like HD (with thousands of affected individuals) and impact the FDA’s thinking on AMT-130.
Two petitions: ‘AMT-130 could change everything’
As of November 15, the two online petitions to the FDA from HD advocates have already garnered almost 9,000 signatures.
These moving, persuasive petitions effectively portray the devastating impact of HD on patients and families and the historic breakthrough towards a treatment achieved with AMT-130. They effectively demonstrate the profound need for the drug and urgent action by the FDA.
One petition is titled “Bring Hope to Huntington's Disease Families: Urge the FDA to Uphold Accelerated Approval.”
It is sponsored by the Huntington’s Disease Society of America, HD Reach, Help4HD International, Huntington’s Disease Foundation, and Huntington’s Disease Youth Organization. These organizations have also pledged to improve collaboration.
It notes that “the FDA is now wavering on its commitment” to AMT-130. It asks the agency to honor its previous guidance to uniQure, recognize the urgency of the unmet dire medical need of HD families, and to expedite the approval of AMT-130.
It urges people to sign: “We cannot allow procedural hesitation to become a death sentence.”
The other petition is titled “Accelerate Breakthrough Drug Approval for Huntington's Disease - UniQure AMT-130.”
“Our Plea: Turn Heartbreak Into Hope,” it states. “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.”
As an HD gene carrier and HD family member, I immediately signed the first petition upon learning about it. I urge all friends and supporters of the HD community to join us in our plea to the FDA.
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