Trump official ousted, FDA reversal clears gene therapy
▼ Summary
– A gene therapy for Huntington’s disease has a new approval path from the FDA after the ouster of Trump officials, including Vinay Prasad, who rejected the therapy.
– Huntington’s disease is an inherited condition with no current treatments, typically striking in middle age and causing brain nerve cell breakdown.
– UniQure’s one-time gene therapy, AMT-130, aims to lower mutant huntingtin protein levels and showed up to 75% disease progression slowing in early trials.
– The FDA initially allowed UniQure to seek accelerated approval without a placebo control, avoiding ethical issues of sham brain surgeries for a control group.
– Under Prasad, the FDA reversed course, demanding sham surgeries as controls, but his ouster has opened a new path for the therapy.
A major reversal at the Food and Drug Administration has breathed new life into a gene therapy for Huntington’s disease, following the removal of several Trump-era officials including Vinay Prasad. One former FDA official described Prasad’s previous rejection of the treatment as “truly evil.”
Huntington’s disease is an inherited neurological disorder that typically emerges in middle age, causing the progressive breakdown of nerve cells in the brain. Currently, no approved treatments exist, and most patients die in their 50s or 60s.
Biotech firm UniQure has developed a one-time gene therapy called AMT-130, designed to reduce brain levels of the mutant huntingtin protein responsible for the disease. Early data from a small trial suggested the therapy could slow disease progression by up to 75 percent, generating intense hope among patients and advocates.
In 2024, the FDA initially told UniQure it could pursue accelerated approval for AMT-130 without including a placebo control group. That decision carried significant ethical weight. Administering AMT-130 requires a 10- to 12-hour brain surgery, meaning a placebo-controlled trial would force control patients to undergo a lengthy sham procedure involving drilling a superficial hole in their skulls.
UniQure moved forward without a placebo arm, relying instead on external, untreated patients as a comparative control group. The company believed the FDA had endorsed this approach. But when Prasad took over as the agency’s top regulator for gene therapies, the FDA abruptly reversed course and demanded that UniQure conduct sham surgeries for its control group. That demand effectively stalled the therapy’s path to approval.
Now, with Prasad and other Trump appointees removed from their posts, the FDA has reversed its stance once again. The agency has cleared the way for UniQure to proceed without sham surgeries, restoring the original agreement and giving the gene therapy a renewed chance at reaching patients who have no other options.
(Source: Ars Technica)
