FDA goes on offense amid surge of criticism for recent drug decisions

By Meg Tirrell, CNN

(CNN) — In a highly unusual move for a normally buttoned-up regulatory agency, a senior official with the US Food and Drug Administration held a call with reporters Thursday to defend the agency’s recent decision on an experimental medicine, calling it a “failed therapy” and railing against outside critics.

The call focused on the FDA’s decision to ask for an additional clinical trial from the biotechnology company UniQure before greenlighting it to apply for approval of its gene therapy for Huntington’s disease, a fatal neurodegenerative disorder. Patients and the company had hoped the therapy could move into an FDA review based on existing evidence, a hope that was dashed Monday.

But the regulator, who spoke on the condition of anonymity, also attacked outside scientists who serve on advisory committees for the agency, saying they “do not do their homework” and may be motivated by financial conflicts of interest. The official additionally singled out Dr. Janet Woodcock, a retired FDA veteran who called the UniQure decision “truly evil” in an interview with the New York Times this week.

“Dr. Woodcock is an esteemed regulator,” the official said, “and as such, there are many commenters who I don’t expect better from, but Dr. Woodcock, I do expect better from.”

Woodcock declined to comment Thursday on the FDA official’s comments, saying she believed that the FDA should review the application and give it a public hearing.

The FDA call represented a tense crescendo in a series of moves that have raised questions about whether the regulator is changing goalposts when it comes to drug approvals.

Numerous companies have claimed that recent actions by the regulator ran counter to prior guidance, slowing approvals of new medicines even as the agency has been touting ways it plans to speed up the process.

Last month, the agency faced a firestorm of criticism for initially refusing to review an application from Moderna for an mRNA flu vaccine before ultimately accepting the application with some adjustments. It held a similar media call then with a different senior official to defend itself.

The FDA has also been criticized for recent moves on rare disease drugs from Atara Biotherapeutics and Pierre Fabre Pharmaceuticals, Regenxbio and others.

On Thursday, the FDA official disputed news coverage and statements from UniQure suggesting that the agency is requiring the company run a clinical trial involving a fake 10-hour brain surgery as a placebo control for the gene therapy, which is given as a one-time infusion administered directly into the brain in an eight- to 10-hour procedure.

“Does the sham intervention require 10 hours of anesthesia? No, it does not,” the official told reporters. “It takes under 30 minutes of anesthesia. … Did FDA ask UniQure to drill holes in skulls? No, they just need to make one to three nicks in the scalp.”

The official said the agency requires placebo-controlled trials to ensure that the product is not “snake oil” and argued that it’s longstanding FDA policy to require them for Huntington’s disease treatments.

Woodcock told CNN that she believes the FDA should review UniQure’s therapy.

“For such a terrible disease and people who volunteered to have this therapy put into their brains, they owe it to the community to review and have a public discussion,” Woodcock said. “They promised ‘radical transparency.’”

She noted that she doesn’t have an opinion on the company’s data itself, as she hasn’t seen it. “This is more about the process.”

UniQure had hoped to make a submission to the FDA using data from an earlier trial that suggested its therapy slowed disease progression compared with an external control group made up of people with Huntington’s who weren’t in the study.

The communications with the agency took place in a routine series of interactions in which the FDA provided guidance on whether UniQure could submit an application for approval with existing data. The company has called the agency’s new posture “a drastic change” from guidance it provided in late 2024.

UniQure said in a statement Thursday, “the recent statements made by anonymous FDA sources to the press have been highly irregular, unprecedented, and are incomplete or entirely incorrect. We do not believe they reflect a fair and faithful reading of the documents we have submitted or those we have received from the agency.”

The company added that it’s “confident in the strength of the data we have submitted to the FDA, and the real potential” for the treatment to benefit patients with Huntington’s disease, and that it hoped to move forward with more direct conversations with regulators.

“We appreciate and strongly share the administration’s goal of developing meaningful treatments for patients suffering from rare diseases,” UniQure said.

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