Prasad's Request To Remain Anonymous Shines Light on FDA's Transparency Problem

While requests by government officials for anonymity when speaking to the media are nothing new, the practice attracts more scrutiny when the Department for Health and Human Services has pledged a commitment to “radical transparency.”

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. I say “now-outgoing” because Prasad’s upcoming departure wasn’t known at the time of last week’s media briefing, though the administration says he had always planned to leave the agency after a year.

For a Department of Health and Human Services (HHS) that has often touted efforts toward “radical transparency” under Robert F. Kennedy Jr., that’s a lot of secrecy.

To be fair, Prasad did not work very hard to conceal his identity, describing himself during the briefing as “a professor” and “practicing [hematology-oncology] doctor,” STAT News reported. Prasad also told select reporters that he missed his clinic and teaching, and the ability to run his podcast and “speak freely.” So, when

news brokethe next day that he would depart the FDA—

for a second time—at the end of April, it was somewhat unsurprising.

It begs the questions, however: If Prasad prefers but was unable to speak freely as director of the Center for Biologics Evaluation & Research and already knew he was out the door, who exactly was behind the anonymity request, and why?

The episode also highlights a continued lack of transparency within the FDA and other HHS agencies, despite the administration’s promises.

While Kennedy and his team of healthcare administrators have promoted such initiatives as the publication of Complete Response Letters for rejected products as “radical transparency,” most new policies have raised more questions than they’ve answered. And then there are the behind-closed-doors discussions, reversals of course on guidance for drug candidates and requests for anonymity involving senior FDA staff.

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.

And of all the things to not be transparent about, the planned one-year tenure of the most controversial CBER director in recent memory—maybe of all time—stands out. Why not let the industry and the American people in on this plan from the beginning? Or was it just an attempt to save face when FDA Commissioner Marty Makary realized he’d have to let Prasad go once and for all?

When Makary told The Wall Street Journal last Friday 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, it was news to me. It also was clearly news to

Bloombergreporter Robert Langreth, who asked Prasad at the briefing the day before how long he planned to stay with the FDA,

STATreported.

This plan was not disclosed when Prasad was brought on to FDA in May of last year, nor was it mentioned when Prasad left the agency in the midst of the Sarepta saga, only to return 10 days later, apparently at Makary’s behest. And Prasad kept this secret even as he responded directly to Langreth’s question, saying that as long as Makary, Kennedy and President Donald Trump wanted him to lead CBER, he would “continue to serve happily,” according to STAT.

I reached out to HHS last Friday seeking a response to this question and followed up again this week. I have yet to receive a response. I have found that this lack of response is, sadly, common these days. And sometimes when I do hear back, the answers to my questions are provided on “deep background”—not to be used in my stories. So much for radical transparency.

An ‘Extraordinary’ Event

The other alarming aspect of the media event, which H.C. Wainwright in an investor note this week called “extraordinary,” was its content. Prasad reportedly accused uniQure of pushing “distorted” and “manipulated” clinical data for a “failed” therapy. This, despite the agency’s previous assertion that it “generally cannot provide confidential information about unapproved products.”

Prasad wasn’t the only one bashing AMT-130. In a late February interview with CNBC, Makary himself appeared to disparage the gene therapy, without naming names.

“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, Makary told the network. “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.”

AMT-130 slowed disease progression by 75% after three years in a pivotal trial, uniQure reported in September. The 12-month sham surgery–controlled cohort of the trial did not show benefit at that time point, however, a fact that analysts H.C. Wainwright noted as the FDA’s primary sticking point. The three-year data was compared to external controls, which the agency has repeatedly taken issue with, despite encouraging their use in recent rare disease guidance.

UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms

Another point of contention between the FDA and uniQure regarding AMT-130 is the sham-controlled Phase 3 trial the agency is insisting upon before it will review AMT-130. This represented a “key shift” from guidance uniQure received from the FDA in 2024, the company has said.

In addition, the nature of the sham surgery the FDA is requesting as the control remains unclear. During the company’s 2025 earnings call on March 2, uniQure Chief Medical Officer Walid Abi-Saab told investors that the trial would require patients to be anesthetized for 10 to 12 hours while a surgeon cuts through the skin and makes a superficial (burr]) hole in the skull. Prasad disputed this, telling select reporters on the media call that uniQure would only need to “put one to three nicks in the scalp.”

That there is any confusion regarding such a critical—and ethically thorny—aspect of the trial is concerning. It also suggests that transparency is sorely lacking in the FDA’s communication regarding this program—transparency not just to media and the public, but to the drug sponsor itself.

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A Radical Lack of Clarity

While uniQure is the most recent point of friction within the FDA, it is merely the tip of the iceberg in terms of transparency issues over the past 12 months.

Biopharma is still seeking clarity around recently announced FDA policies, including the transition from a requirement of two pivotal trials to one for new drug applications and the Commissioner’s National Priority Voucher (CNPV) program, first introduced last summer.

In a letter to the FDA last month, Rep. Jake Auchincloss (D-MA) said the CNPV program was “shrouded in secrecy,” according to reporting from the Associated Press, noting that the agency has failed to respond to several inquiries from Congress about the scheme.

Meanwhile, Harpreet Singh, chief medical officer at Precision for Medicine and a former division director of oncology at the FDA, lamented the current lack of certainty regarding FDA communication.

“I feel that in the macro picture of things, what bolsters innovation is regulatory certainty,” she told me in a recent interview, “and we very much are lacking that right now.”

Notably, the transparency problem is not unique to the FDA. In May 2025, RFK Jr. announced via X that the CDC would remove routine COVID-19 vaccination for healthy children and healthy pregnant women from its guidelines. Kennedy revealed the new policy in a 58-second video posted to social media, neglecting to explain the rationale for the decision to CDC leadership.

“CDC scientists have still not seen the scientific data or justification for this change,” Debra Houry, the agency’s former chief medical officer, told the Senate Committee on Health, Education, Labor, and Pensions in September, when it convened to address the controversial firing of former CDC director Susan Monarez. “Trust and transparency have been broken.”

The uniQure brouhaha has once again brought transparency—or the lack thereof—within RFK Jr.’s HHS to the forefront. In this case, we’ve got the FDA commissioner speaking negatively about the gene therapy without actually naming it, the CBER director explicitly bashing AMT-130 but requesting to remain anonymous while doing so, a reversal of course on previous FDA guidance to the company and utter confusion over uniQure’s path forward.

And perhaps most shockingly, we also have the CBER director at the center of the controversy suddenly announcing his apparently long-planned exit from the agency. Makary claims the plan was always for Prasad to exit the FDA after a year, but Prasad said just the day before that he would continue to serve as long as he was needed. If his departure next month was always Makary’s plan, did anyone bother to tell Prasad?