There was a telling exchange during a Thursday media call set up by the Food and Drug Administration to enable a “senior FDA official” — he could only be quoted anonymously — to attack UniQure and its experimental treatment for Huntington’s disease.
Robert Langreth, a reporter for Bloomberg, asked the senior official how long he planned to stay at the FDA.
The FDA official responded, “You all know that as background, I’m a professor and I’m a practicing [hematology-oncology] doctor. I do miss my clinic and I miss teaching, and I miss being able to run my podcast and speak freely. But I would say that I serve at the pleasure of the [FDA] commissioner and the [health] secretary and the president, and as long as we’re all in alignment — that me serving is in the best interest of the American people — I will continue to serve happily.”
The agency, in other words, handed regulatory oversight for drugs intended to treat rare and deadly diseases to a person who misses their hot-take podcast.
What started months ago as scientific disagreement between the FDA and UniQure over how best to study a gene therapy for Huntington’s disease metastasized on Thursday into a diatribe by the senior agency official, who accused UniQure of pushing “distorted” and “manipulated” clinical data for a “failed” therapy.
STAT Plus: FDA is ‘not convinced’ UniQure’s Huntington’s therapy has benefit, senior official says
It was a performance that might not raise eyebrows on a podcast, but the official was speaking on an FDA-organized media call — a norm-busting decision from an agency that is set up to review clinical data objectively, not lob attacks against the companies it is in charge of regulating.
The comments and behavior of the senior FDA official threaten to turn UniQure into another political headache for the Trump administration as the midterm elections approach.
The administration is trying to shift focus away from controversial health care issues, including vaccine policies, and toward topics that are popular with voters, such as lowering drug prices and encouraging healthy diets, my STAT colleague John Wilkerson wrote recently.
Instead, the FDA has repeatedly found itself the subject of controversy, particularly when it comes to its regulation of rare disease drugs — a sensitive subject politically since President Trump has won praise for championing the rights of patients with rare diseases during his first term.
As Wall Street Journal reporter Liz Essley Whyte noted in a story Thursday, the official’s attacks on UniQure “risk further angering members of Congress who have been pressing the Trump administration to be more open to approving rare-disease therapies, after FDA officials rejected or delayed some applications of some new drugs.”
CNN correspondent Meg Tirrell, in a story Thursday, linked the UniQure situation to last month’s FDA-Moderna flu vaccine episode, in which the agency faced severe criticism for initially refusing to review Moderna’s application but then accepted it with adjustments. The agency held a similar media call with a different senior official at that time, Tirrell noted.
Politico reporters Lauren Gardner and Tim Rohn reported that the FDA’s reversal on Moderna’s flu vaccine followed White House pressure. FDA Commissioner Marty Makary was called to the White House and told by Trump that he was frustrated by the FDA’s handling of vaccine issues.
More criticism of the agency appears to be on the horizon. On Thursday, Sen. Ron Johnson, a Republican from Wisconsin, told my colleague John Wilkerson that he was planning to use public pressure to get the FDA to change its ways on rare-disease treatments. Johnson said he was planning a press conference next week with advocates representing patients with Duchenne muscular dystrophy, and that he had spoken to Makary.
The FDA has showered the politically influential Duchenne community with remarkable generosity in recent years — approvals of a gene therapy and multiple drugs made by Sarepta Therapeutics despite clinical trials that have failed repeatedly to demonstrate the products’ benefit to patients.
The agency’s maximal flexibility in Duchenne stands in stark contrast to the way Huntington’s patients are being treated — refusing to even allow UniQure to file an application and trashing its work anonymously.
