FDA official defends controversial gene therapy decision
In a highly unusual move, a senior official with the US Food and Drug Administration (FDA) held a call with reporters to defend the agency’s decision regarding an experimental gene therapy for Huntington’s disease, branding it a “failed therapy” and challenging external critics, reports BritPanorama.
The FDA requested an additional clinical trial from the biotechnology company UniQure before allowing it to apply for approval of its gene therapy, a pivotal treatment aimed at a fatal neurodegenerative disorder. Many patients and stakeholders had anticipated that existing evidence would suffice for a review, only to see those hopes dashed recently.
During the call, which was conducted under conditions of anonymity, the official took aim at criticisms from scientists serving on advisory committees, accusing them of insufficient diligence and potential conflicts of interest. In particular, the official criticized Dr. Janet Woodcock, a veteran FDA figure, who termed the UniQure decision “truly evil” in a recent interview.
The official remarked, “Dr. Woodcock is an esteemed regulator…there are many commenters who I don’t expect better from, but Dr. Woodcock, I do expect better from.” Woodcock did not provide a comment for the media following the statements.
This public defense from the FDA follows mounting tensions surrounding its regulatory practices, prompting scrutiny over whether it is shifting its criteria for drug approvals. Recent actions by the agency have drawn significant scrutiny, with various companies asserting that FDA decisions contradict previous guidance, thereby slowing the approval process for new treatments, despite the FDA’s stated intentions to expedite these processes.
Last month, the agency faced backlash for its initial refusal to review a submission from Moderna concerning an mRNA flu vaccine, which was later accepted following adjustments. A similar media engagement was held by a different senior official to address the surrounding criticism.
The FDA has recently faced further scrutiny related to approvals for rare disease drugs from companies including Atara Biotherapeutics and Regenxbio. On Thursday, the FDA official contradicted reports stating that the agency requires a clinical trial involving a fake 10-hour brain surgery as a placebo for UniQure’s therapy, clarifying that such an intervention does not necessitate prolonged anesthesia.
“Does the sham intervention require 10 hours of anesthesia? No, it does not,” the official responded. “It takes under 30 minutes of anesthesia.” The official also emphasized the importance of placebo-controlled trials to ensure legitimacy, reiterating that this has been standard FDA policy for Huntington’s disease treatments.
UniQure had aimed to leverage data from an earlier trial indicating that its therapy could decelerate disease progression compared to a control group not involved in the study. However, communications in a series of routine interactions suggested a significant change in the agency’s guidance compared to late 2024.
While UniQure declined to comment on Thursday, earlier this week, its chief executive expressed incredulity at the FDA’s public communications, highlighting a gap in dialogue between the agency and the company.
The unfolding situation underscores growing tensions within regulatory processes as companies navigate complex approval landscapes amidst evolving FDA policies.
