As uniQure seeks to overcome ‘dysfunction’ at the FDA, Huntington’s disease community rallies in defense of gene therapy drug

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.

Understanding the FDA’s surprising step back on uniQure drug that slows Huntington’s disease

 

Less than six weeks after uniQure announced that its gene therapy drug slowed the progression of Huntington’s disease by 75 percent over three years, the U.S. Food and Drug Administration (FDA) has backtracked on its conversations with the company regarding the timeline and data needed for potential approval of the remedy.

 

The company’s plan to apply for approval of the drug, AMT-130, in early 2026 and launch it into the market later that year may no longer be possible.

 

In a November 3 press release uniQure announced that, after a recent meeting with the FDA, the company believes that the “FDA currently no longer agrees” that data from its clinical trial of AMT-130 using an "external control" as a comparison "may be adequate to provide the primary evidence in support of” an application for approval.

 

The external control refers to data taken from Enroll-HD, the global HD patient registry of more than 22,200 people, as a baseline, rather than those taking a placebo, to compare with those on AMT-130. After extensive consultations with the FDA in 2024, uniQure had gotten permission from the agency to use the Enroll-HD data.

 

“This is a key shift from prior communications with the FDA in multiple … meetings over the past year,” the uniQure release stated. “Consequently, the timing of the BLA [Biologics License Application] submission for AMT-130 is now unclear.”

 

uniQure expects to receive the final minutes of its meeting with the FDA within 30 days and plans to urgently interact with the agency “to find a path forward for the timely accelerated approval of AMT-130,” the press release continued.

 

“We are surprised by the FDA’s feedback at the recent pre-BLA meeting, which is a drastic change from the guidance the FDA provided in November 2024,” said Matt Kapusta, the uniQure CEO. “This news is unexpected, and we are truly disappointed for people living with HD, who have no disease-modifying treatment options for this devastating disease.”

 

Kapusta added that “we strongly believe that AMT-130 has the potential to bring substantial benefit to patients, and we remain fully committed to working with the FDA to determine the best path forward to rapidly bring AMT-130 to patients and their families in the U.S.”

 

 

David Margolin, M.D., Ph.D., uniQure's vice president for clinical development, presents data illustrating AMT-130's slowing of the progression in Huntington's disease at the 20th Annual HD Therapeutics Conference, Palm Springs, CA, February 25, 2025 (photo by Gene Veritas, aka Kenneth P. Serbin).

 

FDA claims to ‘unleash gene therapies’

 

The October 30 Huntington’s Disease Society of America (HDSA) webinar on AMT-130, featuring uniQure’s chief medical officer, Walid Abi-Saab, M.D., offered no inkling of the FDA’s new position.

 

Some 1,000 people attended the webinar – a sign of the excitement about AMT-130 in the HD community. Reuniting after 17 years of estrangement, my own family has found great hope in the possibility of this treatment, though we understand that the drug is not a cure and might not even reach the public.

 

The FDA so far has provided no comment to the media on the matter. At the top of its website’s homepage it has the phrase “first six months of FDA reforms,” including “unleashing cell and gene therapies.”

 

As this blog reported in July, under the Trump administration the uncertainty of public funding for science has created serious challenges to HD research.

 

Politics and society play a role

 

Science and medicine do not act in a vacuum and are impacted by politics and society, as HD family member and historian Alice Wexler brilliantly illustrated in her book The Woman Who Walked into the Sea: Huntington’s and the Making of a Genetic Disease (click here to read my review).

 

In July, a New York Times report titled “Inside the Collapse of the FDA” detailed how Health and Human Services Secretary Robert F. Kennedy Jr., is “dismantling the agency.”

 

The key biotech and life sciences website STAT reported that since uniQure and the FDA had set drug submission “benchmarks” last year, “the agency has undergone considerable changes. Vinay Prasad, a physician with a reputation for taking a more conservative view of gene therapies, now oversees the division with authority over AMT-130.”

 

Dr. Prasad heads the Center for Biologics Evaluation and Research.

 

Only last week STAT reported that a “slow-boiling feud” between Dr. Prasad and his staff “is threatening the future of the center that regulates the nation’s vaccines, biological products, and blood supply.”

 

According to the report, officials in the center described it as “rife with mistrust and paranoia” and with fears of “retaliation” for pushing back on Dr. Prasad. The center has lost hundreds of employees this year to retirements, layoffs, and resignations. Dr. Prasad has also pushed out senior leaders, including “top cell and gene therapy regulators,” STAT reported.

 

Prasad has also played a role in limiting access to Covid-19 vaccines, according to STAT and the New York Times.

 

The HD community is ‘highly mobilized’

 

The scientist-written website HDBuzz noted that, although “the reason for the change of heart” of the FDA is “not currently clear,” “it makes the timeline for advancing AMT-130 less clear, with additional uncertainty as the U.S. government shutdown continues.”

 

“The decision by the FDA to not agree that an external control group can be used to apply for a [drug application] doesn’t change the data” of AMT-130’s actual effect in the clinical trial run by uniQure, HDBuzz stated. It simply means that the FDA would want to see more data, possibly from a trial designed with a placebo control group, before moving forward with a BLA for AMT-130.

 

HDBuzz also noted that uniQure is working with the equivalent of the FDA in the United Kingdom and Europe, with an approval there possibly leading to acceptance elsewhere.

 

HDBuzz stated that “we push forward – together.”

 

On November 5 HDSA and four other HD entities issued a joint statement: following the regulatory update about uniQure, “it’s clear that stronger alignment and collaboration among Huntington’s disease (HD) patient and family organizations are more critical now than ever.” The four are Huntington’s Disease Foundation (formerly Hereditary Disease Foundation), HD Reach, Help4HD International, and Huntington’s Disease Youth Organization.  

 

“The next steps here are uncertain,” the brokerage and investment banking firm Stifel wrote, “but given the highly mobilized patient community in Huntington’s, and other external political forces that may have influence on the agency, the story here may not be over.”