uniQure announced on January 9 that it has scheduled a high-priority meeting with the U.S. Food and Drug Administration (FDA) to revisit its application to seek approval of its gene therapy drug for Huntington’s disease, after the agency had rolled back its permission.
As recounted here, uniQure and others in the frustrated biotech sector believe that the FDA has become dysfunctional under the Trump administration (click here and here for details).
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, in November uniQure announced that the FDA had abruptly switched gears regarding its agreed-to plan for considering AMT-130.
In its January 9 press release, uniQure stated that it had scheduled a so-called Type A meeting with the FDA “to support accelerated approval of AMT-130.” It did not disclose the date of the meeting.
“We look forward to a constructive discussion with the FDA as we work toward a timely resolution regarding an accelerated approval pathway for AMT-130,” stated uniQure CEO Matt Kapusta. “The Huntington’s disease community, including patients and clinicians, has emphasized the profound unmet medical need and the importance of timely access to potentially disease-modifying therapies such as AMT-130. We remain deeply committed to patient access while continuing to collaborate closely with the FDA.”
Seeking to resolve the dispute over AMT-130
As noted by the scientist-written website HDBuzz, Type A FDA meetings “occupy a special category.”
“They’re reserved specifically for situations where a development program has stalled, like when there’s a clinical hold, major safety concerns, or formal dispute that need to be addressed before a drug can move forward,” HDBuzz explained.
Type A meetings are scheduled within 30 days of a request, HDBuzz stated, adding that a Type A meeting can help to “resolve disputes” about clinical trial design or regulatory decisions, “chart a path forward,” and “clarify expectations” so that firms know precisely what the FDA requires. (Click here for the FDA’s official explanation of its meeting types.)
Based on this timeline, news from the meeting could be reported in late January or February. The uniQure release stated that the company would provide an update after receiving official minutes from the FDA.
Pressing our leaders
As of January 12, nearly 47,000 individuals had signed the two petitions from the HD community urging the FDA to expedite approval of AMT-130.
The Huntington’s Disease Society ofAmerica also has urged advocates to contact U.S. representatives and senators to “back fair review of AMT-130” by the FDA (click here to write your representatives).
In addition to signing one of the petitions and writing to Congress, I will call my Senators and Representative to explain the importance of this cause for the HD community and beyond.
I urge everyone to press our political leaders on AMT-130.
As I wrote last month, “the FDA should be on the right side of history in this urgent fight to end devastating diseases.”
