Dr. Vinay Prasad is out at the Food and Drug Administration for the second and hopefully last time within the past 12 months. The controversial drug regulator had placed multiple stumbling blocks before patients seeking care for grave and rare genetic conditions. Let us hope the FDA’s culture changes with his exit.
Among the more troubling stories during Prasad’s tenure concerned the gene therapy Elevidys. In January 2026, the drugmaker Sarepta Therapeutics published results from its EMBARK trial, which evaluated the effects of Elevidys in treating Duchenne Muscular Dystrophy (DMD). Few ailments are more wretched: Young boys lose their muscle function as the disease progresses and eventually need wheelchairs, often before they are teenagers. Most DMD patients die before the age of 25. The trial demonstrated that ambulatory boys with DMD maintained far better motor function than a comparable control group; three years post-treatment, the boys taking Elevidys were doing dramatically better than their untreated counterparts.
This did not seem to matter to Prasad. In July 2025, while leading CBER, he halted shipments of Elevidys, citing patient deaths related to the treatment. This was needlessly alarmist: Nearly 1,000 patients have been treated with Elevidys since its approval. In 2025 two patients died, both of whom had been unable to walk at the time of receiving the therapy. All patients who were ambulatory at the time of receiving the treatment improved as a result. Sarepta concluded that one of those two deaths was unrelated to Elevidys. A third death cited by the FDA in its July decision to suspend the distribution of Elevidys was unrelated to the trial entirely: that patient was a 51-year-old man with a different condition who had taken a different experimental Sarepta drug.
The FDA first granted accelerated approval for Elevidys back in 2023, with the understanding that potentially effective treatments for this well-documented, swiftly moving illness warranted special urgency. But Sarepta and its investors have been in limbo ever since, subject to unpredictable moments of hesitation by an FDA that is susceptible to cold feet, expressed in the form of requests for additional testing or distribution pauses.
Elevidys works by delivering a functional gene to muscle cells that enables production of a shortened “micro-dystrophin” protein, which substitutes for the missing dystrophin in DMD. It is administered as a single intravenous infusion, addressing the root genetic cause of DMD in a way no standard drug can. The ethics of including a control group in a trial for a well-documented disease aside, these results ought to have been grounds for celebration. Yet the FDA, under the guidance of Prasad, continued to move the goalposts, making the lives of patients and the work of drugmakers ever more burdensome.
Prasad was swiftly lambasted by the Wall Street Journal’s editorial board for attempting “to kill a therapy that has helped boys with a deadly disease.” At the time, he briefly left his post before returning less than two weeks later.
During his tenure, Prasad—an avowed champion of both Bernie Sanders and “a strong regulatory state”—repeatedly sought to foil attempts by desperately ill young men to access treatment. After the outrage at Prasad’s decision to withdraw Elevidys, outrage from the families of children with DMD forced the agency to reinstate it. But in November, Prasad insisted on attaching a safety warning letter to Elevidys, which highlights potential dangers in a fatalistic way. It is but one example among many in which Prasad appears more fearful of gene therapies than of the frightful, and often rare, conditions they are designed to treat.
This is not the first time boys with DMD have been locked in regulatory warfare, nor is it the first time the Wall Street Journal has come to their defense. In April 2016, a 13-member FDA advisory committee voted against approving Eteplirsen, a different Sarepta drug for DMD. Eteplirsen had been tested on 12 boys around the age of ten, all of whom still walked after four years on the drug. Only one boy in an untreated control group could still walk at the end of the trial. Not one of the children who had been given the drug has suffered any serious side effects. Nevertheless, the FDA committee decided that 12 patients were too few to draw meaningful conclusions.
As the Journal noted at the time, this was “a convenient flip from 2014, when FDA said it’d consider a patient-comparison trial in one of a dozen meetings with Sarepta.” It also noted that Sarepta was a small shop and “couldn’t summon the resources to produce unlimited drug doses at government whim.”
Much of the FDA reporting on DMD treatments has focused on ancillary arguments: who told Sarepta what, and when, or whether Sarepta reported the deaths of two patients in a sufficiently public and timely fashion. Less noted, however, is the FDA’s culture of ignoring or denying its responsibility: When the FDA denies potentially transformative treatments to DMD sufferers who want them, the FDA becomes responsible for the worsened suffering they experience as a result, and the potential for earlier death that comes along with the condition.
Prasad’s public writing shows he did not think this way. In a 2021 paper about an FDA-approved cell therapy for multiple myeloma—a treatment that reduced disease progression or death by half among patients with advanced cancers who hadn’t responded to already approved therapies—he claimed “it is crucial to question whether non-curative therapies . . . are worth it.” The therapy was expensive and might “only delay inevitable progression” of the disease in some patients. Such logic is, of course, absurd: any number of therapies, from Tylenol to cancer treatments, are merely meant to prolong the inevitable progression of the human life cycle, which ends in death.
Prasad often seemed almost in a hurry to help shuffle the afflicted off the mortal coil. In a 2022 paper, Prasad estimated that patients with advanced cancer spend 16 more hours a month accessing and receiving novel treatments than if they had accepted hospice or home palliative care. “Time is a valuable resource for people who have cancer,” Prasad noted, without considering why such patients are willing to gamble to get more of it. In 2017, he authored an op-ed lauding the highly restrictive approach to drug research in the United Kingdom and suggested the U.S. consider doing more to ration treatments. Here too, Prasad ignored the fact that survival rates for hard-to-treat cancers are much lower in the U.K. than in America, and that British patients often travel abroad in search of life-saving medicine. In these and many other instances, Prasad advises patients and drug designers simply to throw in the towel and save us all some time and money.
Of course, Prasad's actions at the FDA decisions were taken under Commissioner Martin Makary’s leadership. Prasad’s departure is welcome, but it does not resolve the deeper problem. The regulatory culture that produced these decisions took hold under Makary and will persist unless leadership at the agency changes course.
Trump’s regulatory team ought to reject this way of thinking about human life and cost-benefit analysis. It ought to take responsibility for the consequences not only of the drugs it approves, but of those it rejects. And it should recognize the value of giving patients choices, and drugmakers the incentive to produce new and better medicines.
