A gene therapy for Huntington’s disease has a new path toward approval from the Food and Drug Administration after the ouster of several Trump officials, particularly Vinay Prasad, who rejected the therapy in a shocking move one former FDA official called “truly evil.”
Huntington’s disease is an inherited condition that typically strikes in middle age and causes nerve cells in the brain to gradually break down. There are currently no treatments for the disease, and many afflicted die in their 50s and 60s.
Gene therapy company UniQure developed a one-time treatment, AMT-130, that aims to lower brain levels of the mutant protein behind the disease, called huntingtin. Data from a small, early trial suggested the drug could slow the progression of the disease up to 75 percent, and patients and advocates have closely watched the drug’s development in hopeful anticipation.
In 2024, the FDA indicated to UniQure that it could file for accelerated approval of AMT-130 without a placebo control arm in its trial. While having a placebo control offers a high-quality comparator in a trial, it raises unique ethical concerns for UniQure’s gene therapy. Delivery of AMT-30 requires a 10- to 12-hour brain surgery, which means a placebo-control arm of a trial would require patients in a control group to undergo a lengthy sham surgery that could involve drilling a superficial hole in their skulls.
UniQure moved forward without a placebo control, using external, untreated patients as a comparator control group for their trial, believing the FDA backed the plan. But during Prasad’s tenure as the FDA’s head regulator of gene therapies, the agency tossed the agreement and demanded that UniQure conduct sham surgeries as controls.
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