On November 2 uniQure announced that the U.S. Food and Drug Administration (FDA) has accepted its application seeking approval of its gene therapy drug, which has shown historic efficacy in slowing the progression of Huntington’s disease.
The therapy AMT-130, now officially known as ifezuntirgene inilparvovec (eye-feh-ZUN teer-JEEN in-il-par-VOE-vek), demonstrated 75 percent slowing of HD in clinical trial results released in 2025. Last month, additional trial data revealed slowing of 44 percent, which was still seen as substantial by researchers.
According to the uniQure press release, ifezuntirgene inilparvovec will receive priority review, with May 1, 2027, as the target date for the FDA’s approval decision.
“The FDA’s acceptance of our BLA [Biologics License Application] for review marks an important milestone for the Huntington’s disease community, which has waited generations for
a treatment that changes the course of this devastating disease," CEO Matt Kapusta stated in the release. "Backed by compelling data from our Phase I/II studies, we believe ifezuntirgene inilparvovec has the potential to become the first disease-modifying treatment for Huntington’s disease.”
A new clinical trial to buttress the data
By the end of 2026, uniQure expects to begin screening volunteers in the U.S. for a required confirmatory clinical trial involving 200 people. Sites in other countries will be announced later. The trial will be “well underway” by May 1, Kapusta added.
The acceptance comes after the dysfunctional FDA under the Trump administration broke its promise to let uniQure submit an application in early 2026, prompting HD advocates to file two petitions with the agency having more than 48,000 signatures. In May, that crisis helped provoke the resignation of FDA Commissioner Marty Makary, leading uniQure in June to announce a new plan for drug approvals (click here to read more).
Recognizing the urgent need for rare disease treatments, the FDA is intensifying its efforts to foster drug development for those conditions.
The Rare Disease Framework Initiative
In an October 14 press release the U.S. Department of Health and Human Services (HHS), which oversees the FDA, announced “the Rare Disease Framework Initiative to reimagine the development of drugs and biologics for rare diseases.”
According to the release, the initiative “will develop an optimized, fit-for-purpose framework that reflects the realities of rare disease drug development by considering the nuances of each disease, patient needs, product type and the evidence that can be realistically generated in small patient populations.”
The Initiative will be led by the FDA’s Rare Disease Innovation Hub, partnering with the Office of the Commissioner, the agency’s Center for Biologics Evaluation and Research, and its Center for Drug Evaluation and Research.
Building a more consistent and predictable approach
The initiative addresses concerns HD researchers have long invoked as essential for developing drugs like ifezuntirgene inilparvovec.
“Americans with rare diseases cannot afford a regulatory system that treats every condition the same,” the highly controversial HHS Secretary Robert F. Kennedy, Jr., who tried to dismantle the agency, stated in the HHS release. He added that the FDA is “rethinking rare disease drug development by putting patients at the center and matching regulatory requirements to the realities of small patient populations.”
“Patients and families living with rare diseases have few or no approved treatment options, and the burden of uncertainty can extend across every part of their lives,” Acting FDA Commissioner Kyle Diamantas stated in the release. “We must ensure that America’s regulatory system remains at the forefront — prepared to respond to emerging science, anticipate the needs of patients and families, and help bring safe and effective therapies to those who need them.”
The FDA will build “a more consistent, predictable, and innovative approach to rare disease drug development,” Diamantas added.
On September 24, Heidi Overton, President Trump’s controversial nominee to head the FDA, appeared before the U.S. Senate’s Health, Education, Labor and Pensions committee, facing pointed questions from senators. The full Senate has not yet voted on the nomination.
People ‘genuinely invested’ in rare diseases
The response to the initiative has been positive. On October 28, the FDA held a public hearing to discuss the Rare Disease Framework Initiative at agency headquarters in Silver Spring, MD. Diamantas was present. FDA staffers recognized the importance of providing the regulatory predictability and clear guidance biotech firms need to develop rare disease drugs.
Help4HD International advocate Lauren Holder, like me an HD gene carrier, attended the meeting in person, with several other advocates participating via Zoom. Advocates for other rare diseases also took part.
“I had the incredible honor of attending and speaking at the FDA's Rare Disease Framework Public Meeting,” Holder wrote on Facebook. “You could see and feel the excitement, hope, and passion coming from the rare disease community. But what moved me just as much was witnessing what I believe to be a genuine passion and commitment from the current FDA administration to making this initiative successful.”
In the past, Holder experienced the “heartbreak and frustration” of feeling as if the voices of the HD community “simply didn't matter. Experiences like that can make it incredibly difficult to trust the very system you're fighting so hard to work with.”
This time, however, felt “different,” Holder added. “I didn't just hear promises about innovation, flexibility, and putting patients first. I saw people who appeared genuinely invested in making those things a reality. I felt a willingness to listen, to engage, and to recognize that the rare disease community deserves better.”
Currently, inclusion of the patient perspective in rare disease drug applications is optional. Under the new framework, the FDA will expect it to become an essential part of applications.
Until December 11, anyone can submit a comment to the government regarding the Rare Disease Framework Initiative by clicking here.
The power of solidarity and sharing our stories
In a November 4 press release, uniQure presented its third quarter 2026 financial results and company update. With a total of $768.4 million, the company has continued to accumulate funds, which, according to the release, could support operations into 2030.
In addition to its HD gene therapy, uniQure also has clinical trial programs for mesial temporal lobe epilepsy and Fabry disease.
As science advances, the number of rare diseases discovered keeps growing.
According to the FDA, “An estimated 10,000+ rare diseases affect more than 30 million people – approximately one out of every 10 people – in the U.S., and about half of these people are children.”
Regarding the FDA and rare disease, Holder wrote that she now has “renewed hope and a reason to believe that meaningful change really is possible.”
For the HD community and beyond, her testimony demonstrates the power of solidarity and sharing our stories with the world.
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