“Truly evil” FDA rejection of gene therapy overturned after Trump official ousted

A promising gene therapy aimed at treating Huntington’s disease has received renewed hope for FDA approval following the departure of several officials from the Trump administration, notably Vinay Prasad, who had previously denied the therapy’s advancement. Huntington's disease is a hereditary condition that often manifests in middle age, leading to the gradual deterioration of brain nerve cells. Currently, there are no available treatments, and many patients succumb to the disease in their 50s and 60s. Developed by UniQure, the one-time gene therapy known as AMT-130 is designed to reduce levels of the harmful mutant protein, huntingtin, in the brain. Initial data from a small clinical trial indicated the potential for this therapy to slow disease progression by as much as 75%. As a result, patients and advocates have been closely monitoring its development with a sense of cautious optimism. In a significant shift, the FDA has indicated that UniQure may now file for accelerated approval of AMT-130 without needing a placebo control arm in its trials. While placebo controls are typically considered the gold standard for clinical trials, they posed ethical challenges for this particular gene therapy. The administration of AMT-130 necessitates a complex 10- to 12-hour brain surgery, making the use of sham surgeries for a control group both impractical and ethically questionable. Despite initially having the FDA’s agreement to proceed without a placebo control, the agency under Prasad’s leadership reversed its stance, insisting that the company conduct sham surgeries as part of their trial requirements. This latest development marks a crucial turning point for UniQure and those affected by Huntington’s disease, as they move closer to potentially transformative treatment options.