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

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.

Prasad’s time in the role was generally marked by reports that he overruled staff scientists and career officials, blindsiding companies and moving goalposts.

Back on track

As Prasad continued to double down on the new requirement for UniQure, Janet Woodcock—a retired FDA official who was with the agency for nearly 40 years—blasted the move, calling it “truly evil.” Prasad then lashed out at UniQure and Woodcock in an extraordinary press briefing, calling AMT-130 a “failed therapy,” accusing the company of manipulating data and saying he “expect[s] better” from Woodcock.

A day after that press briefing, then-FDA Commissioner Marty Makary announced that Prasad would depart the agency by the end of April. Makary and another Trump official, Tracy Beth Høeg—who was the top drug regulator—have also since departed amid intense controversy.

In a press release today, UniQure said that in a recent meeting with the FDA, the agency once again agreed that the company could move toward accelerated approval with trial data that includes a control group composed of patients receiving standard care, not sham surgeries. The company expects to file for accelerated approval in the third quarter of this year.

“Today’s announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA’s genuine commitment to addressing the unmet need of Americans living with Huntington’s disease,” UniQure CEO Matt Kapusta said in a statement. “We remain focused on bringing AMT-130 to patients and families as quickly and responsibly as possible in the US and globally.”

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