AI Generated Image
"Truly evil" FDA rejection of gene therapy overturned after Trump official ousted
Ars Technica • • general
Key Points:
- UniQure’s gene therapy AMT-130 for Huntington’s disease, which showed promise in slowing disease progression by up to 75%, has regained a pathway toward FDA approval after earlier regulatory setbacks.
- The FDA under Vinay Prasad had demanded sham brain surgeries for placebo controls in trials, a move criticized for ethical concerns and later reversed following his departure from the agency.
- Former FDA official Janet Woodcock condemned Prasad’s stance as “truly evil,” and Prasad’s contentious actions, including public criticism of UniQure and Woodcock, preceded his exit from the FDA.
- The FDA has now agreed that UniQure can pursue accelerated approval using trial data with an external control group receiving standard care, avoiding the need for sham surgeries.
- UniQure plans to file for accelerated approval of AMT-130 in the third quarter of 2024, aiming to provide a much-needed treatment option for patients with Huntington’s disease.
