Outgoing FDA Official Anonymously Criticized Huntington's Drug

Vinay Prasad's comments about uniQure's AMT-130 gene therapy raise transparency concerns at the FDA

Mar. 13, 2026 at 2:53pm by Ben Kaplan

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A 'senior FDA official' last Thursday went on the attack against uniQure's investigational Huntington's disease treatment when speaking with press on the condition of anonymity. The official was soon revealed to be the now-outgoing biologics chief Vinay Prasad, who accused uniQure of pushing 'distorted' and 'manipulated' clinical data for a 'failed' therapy. This episode highlights a continued lack of transparency within the FDA and other HHS agencies, despite the administration's promises of 'radical transparency'.

Why it matters

Requests from agency officials to remain anonymous are nothing new, but transparency is even more critical at a time of major policy shifts and constant leadership turnover at the FDA. The planned one-year tenure of the controversial CBER director Vinay Prasad was not disclosed, raising questions about the agency's communication with the public and industry.

The details

Prasad, who is set to depart the FDA at the end of April, described himself during the briefing as 'a professor' and 'practicing [hematology-oncology] doctor.' He told select reporters that he missed his clinic and teaching, and the ability to run his podcast and 'speak freely.' The FDA commissioner, Marty Makary, later revealed that Prasad had only planned to remain at the FDA for the duration of a one-year leave of absence from the University of California, San Francisco, which was news to reporters. Prasad's anonymous criticism of uniQure's AMT-130 gene therapy, despite the FDA's previous assertion that it 'generally cannot provide confidential information about unapproved products,' has also raised concerns about transparency.

  • Prasad went on the attack against uniQure's Huntington's disease treatment in a media briefing last Thursday.
  • Prasad's upcoming departure from the FDA was announced the next day on Friday.

The players

Vinay Prasad

The now-outgoing director of the Center for Biologics Evaluation & Research (CBER) at the FDA, who criticized uniQure's Huntington's disease drug AMT-130 in an anonymous media briefing.

Marty Makary

The FDA Commissioner, who revealed that Prasad had only planned to remain at the FDA for a one-year leave of absence, contradicting Prasad's own statements.

uniQure

The biopharmaceutical company developing the investigational Huntington's disease gene therapy AMT-130, which was criticized by the anonymous FDA official.

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What they’re saying

“There was a product where the researchers drilled a burr hole, literally a hole, in people's skulls' to directly inject a drug candidate into patients' brains. At the end of the randomization period, it was found no benefit, and yet this is one of the drugs that we were pressured to approve.”

— Marty Makary, FDA Commissioner (CNBC)

“We must not let individuals continue to damage private property in San Francisco.”

— Robert Jenkins, San Francisco resident (San Francisco Chronicle)

What’s next

The judge in the case will decide on Tuesday whether or not to allow Walker Reed Quinn out on bail.

The takeaway

This case highlights growing concerns in the community about repeat offenders released on bail, raising questions about bail reform, public safety on SF streets, and if any special laws to govern autonomous vehicles in residential and commercial areas.