A gene therapy for Huntington’s disease has regained its path toward FDA approval following significant leadership changes at the agency. The reversal comes after the departure of official Vinay Prasad, whose previous rejection of UniQure’s treatment was widely criticized within the medical community.
Huntington’s is a fatal inherited condition causing progressive brain cell breakdown with no current cure. UniQure developed AMT-130, a one-time gene therapy targeting the mutant huntingtin protein. Early trial data suggests the treatment could slow disease progression by up to 75 percent.
The regulatory dispute centered on clinical trial design. In 2024, the FDA initially agreed that UniQure could seek accelerated approval using external controls rather than a placebo arm. This waiver was critical because AMT-130 requires invasive brain surgery, and a placebo group would have necessitated unethical sham surgeries involving skull drilling.
Despite this prior agreement, the agency later demanded sham surgeries as controls during Prasad’s tenure. That mandate has now been rescinded, allowing UniQure to proceed with its original trial protocol and offering renewed hope for patients facing this devastating diagnosis.