"Truly evil" FDA rejection of gene therapy overturned after Trump official ousted
Summary
A gene therapy for Huntington’s disease, called AMT-130, now has a clearer path to approval by the FDA after several Trump administration officials who opposed it left the agency. The FDA has agreed to let the company UniQure seek accelerated approval without requiring patients in the trial to undergo risky sham brain surgeries.Key Facts
- Huntington’s disease is an inherited brain disorder that causes nerve cells to break down, with no current treatments.
- UniQure developed AMT-130, a gene therapy aiming to reduce the harmful protein causing Huntington’s disease.
- Early trial data suggested AMT-130 could slow the disease’s progression by up to 75%.
- The FDA originally allowed UniQure to test the therapy without a placebo control group due to ethical concerns about sham brain surgeries.
- Under Vinay Prasad, a Trump-appointed FDA official, the agency reversed this and demanded sham surgeries as controls in trials.
- Prasad’s approach was criticized by other FDA officials, including Janet Woodcock, who described the reversal as “truly evil.”
- After Prasad and other Trump officials left the FDA, the agency returned to supporting UniQure’s plan without sham surgeries.
- UniQure plans to file for accelerated approval in the third quarter of 2024.
Read the Full Article
This is a fact-based summary from The Actual News. Click below to read the complete story directly from the original source.