Even as uniQure, in a December 4 press release, has reconfirmed – based on meeting minutes – the decision by the U.S. Food and Drug Administration (FDA) to roll back its permission to apply for a Huntington’s gene therapy drug approval, more than 43,000 people have signed two petitions demanding the agency to uphold its original plan.
As recounted here, uniQure and others in the frustrated 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 – a historic first.
Then, on November 3, uniQure announced that, after its October 29 meeting with the FDA, the agency had abruptly switched gears regarding AMT-130.
On December 8, the life sciences hub BioSpace reported that “rare disease leaders” want “regulatory consistency” after a “chaotic year” at the FDA.
Overcoming the ‘whiplash of the FDA’s about-face’
The key biotech site STAT has continued to report on the crises in leadership and turmoil at the FDA.
As of December 9, two online petitions to the FDA from HD advocates have garnered more than 43,000 signatures.
Click here and here for the petitions.
In the words of the scientist-written site HDBuzz, “The HD community has not remained silent through the whiplash of the FDA’s about-face from just five months prior, when they stated that data from the ongoing trials would be sufficient to support accelerated approval.”
Honors for AMT-130 researcher
“We are committed to collaborating with the FDA to advance AMT-130 to patients and their families as rapidly as possible,” CEO Matt Kapusta stated in the company’s December 4 release. “The support we have seen these last weeks from the Huntington’s disease community, including patients, families, caregivers, clinicians and advocates, reinforces the urgency of the unmet need in Huntington’s disease.”
Further validation of uniQure and the AMT-130 clinical trial came on December 8, when the key journal Nature announced that Sarah Tabrizi, M.D., Ph.D., a leading HD specialist at University College London and one of the medical leaders of the AMT-130 clinical trial, is “part of Nature’s 10, a list of people who shaped science in 2025.”
Titled “Sarah Tabrizi, Huntington’s hero,” the article about her describes her decades-long efforts to treat HD.
“I want to see if we can prevent Huntington’s from ever occurring,” Dr. Tabrizi told Nature.
As we approach the holiday season and the hopes that HD advocates will persuade the FDA to resume its support of the agreed-to plan for AMT-130, the recognition of Dr. Tabrizi and the community’s massive efforts signal that the FDA should be on the right side of history in this urgent fight to end devastating diseases.
Dr. Sarah Tabrizi at the 2020 Huntington's Disease Therapeutics Conference, Palm Springs, CA (photo by Gene Veritas, aka Kenneth P. Serbin)
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