Dr. Vinay Prasad
His crime? Late last week, the recently appointed chief science officer at FDA had the temerity to tell Sarepta Therapeutics to stop shipping patients its gene therapy for a rare muscle-wasting disease called Duchenne muscular dystrophy, a genetic disorder that manifests in toddler boys and kills most by the time they are young adults. Several reports of sudden deaths among young patients taking the therapy triggered the recall.
It was the right move. An early version of the drug that treated some Duchenne patients had failed in its final efficacy trials submitted to the FDA in 2016. But Dr. Janet Woodcock, then head of the Center for Drug Evaluation and Research, overruled the scientific staff’s negative recommendation to approve the gene-based therapy.
Last year, Drl Peter Marks, head of the Center for Biologics Evaluation and Research, approved a later iteration of the gene therapy called Elevidys that was designed to help all Duchenne patients. He ignored internal staff recommendations against approval by citing improvements in secondary endpoints, anecdotal evidence, and patient desperation. The company never completed clinical trials showing better evidence of efficacy for its primary endpoint.
Marks was forced out earlier this year after Robert F. Kennedy Jr. took over at the Health and Human Services Department. That gave Prasad his chance to assume the reins at CBER.
While early in his career Prasad exposed conflicts of interest in medicine, attacked the paucity of evidence behind new cancer drugs, and blasted the FDA’s decisions approving Sarepta’s gene therapies; the Covid pandemic pushed his contrarian attitudes in a rightward direction. He used his platform on the Sensible Medicine Substack to rail against lockdowns and a broadly administered Covid vaccine, which helped him win a position at FDA under new commissioner Dr. Marty Makary.
The new leadership at FDA must have thought ordering the withdrawal of Elevidys was a no brainer. No proven benefits. Obvious harms. Given they had been raising safety questions about vaccines (without much evidence) to please their new boss at HHS, they were probably caught off guard by the firestorm their decision generated.
“Right to try”
For decades, the most conservative wing of the Republican Party has been pushing what is known as “right to try” legislation. Patients and their doctors, the argument goes, should be allowed to take experimental drugs despite the absence of evidence they work or concerns they cause harm. This movement works hand-in-glove with industry efforts to relax safety and efficacy standards. Rupert Murdoch’s Wall Street Journal editorial page is a vociferous backer of “right to try.”
A limited version of “right to try” legislation passed during Trump’s first term. But the Makary/Prasad move on Sarepta’s gene therapies gave proponents of “right to try” the opening they needed to get rid of Prasad and send Makary a clear message about where his priorities need to be if he wants to stay atop the agency.
It began 10 days ago with a long screed by Trump whisperer Laura Loomer attacking Prasad. His “political alignment is unequivocally progressive, rooted in admiration for far-left figures like Elizabeth Warren and Bernie Sanders. His public statements and voting history reveal a deep-seated opposition to Trump and his policies, making his appointment to a key FDA role baffling,” she wrote.
Then Bob Goldberg, who resuscitates a conservative non-profit called the Center for Medicine in the Public Interest whenever the drug or food industries want to see progressive policies reversed (we frequently crossed swords when I worked for the liberal Center for Science in the Public Interest), weighed in. He attacked Prasad in an op-ed in RealClearHealth: “The 2018 law wasn’t just policy; it was a declaration that in America, a desperate parent has more standing than a bureaucratic panel. That a patient facing death deserves a shot at hope—even if the clinical endpoint isn’t yet statistically significant.”
The Wall Street Journal added its voice to the chorus on Sunday with a long editorial blasting the decision to halt distribution of Sarepta’s gene therapy. It’s worth quoting because of what it portends for future regulatory policy when it comes to new drug approvals:
“This is a regulatory mugging… Vinay Prasad, director of the FDA’s Center for Biologics Evaluation and Research, has lambasted his predecessor Peter Marks for twice overruling agency staff to approve the Duchenne therapy. Dr. Marks made good decisions to prioritize speed and patient choice over certainty on the data.” (Emphasis added.)
Its editorial page was gleeful late yesterday afternoon after the agency reversed the decision halting shipments. “Now the onus is on the agency to show it won’t repeat this debacle and will encourage the innovation that leads to faster cures.”
I assume that includes the unproven innovations that lead to more dead bodies among people receiving therapies that have not yet been shown to work. A few hours later, Prasad was fired.
The takeaway
I shed no tears for Prasad’s short career in public service. Let his experience be a lesson to anyone who thinks agreeing on one issue with the folks running the Trump regime gives you operating room to make decisions that you think are in the best interests of patients and in line with the science, particularly if those decisions are at odds with the financial interests of the biotech and pharmaceutical industries.
Sarepta’s sales of Elevidys and related Duchenne therapies soared to $1.9 billion last year despite fewer than 1,000 patients being served, a reflection of its outrageously high price tag (over $3 million per treatment). Every bit of the R&D that went into the initial development of this particular gene therapy was done with non-profit and government funding.
The patents on the technology were licensed from Nationwide Children’s Hospital in Columbus, OH, which conducted its research with funding from the Muscular Dystrophy Association and the National Institutes of Health. Sarepta’s chief science officer, Louise Rodino-Klapac, did much of her early work on gene therapy for Duchenne’s at Nationwide with NIH grants.
The stock market is clearly pleased by this latest turn of events in the Sarepta saga. Its stock is up over 14% today to more than $18 a share. But that’s a far cry from the $173 a share it traded at a year ago.
Just like its stock price, the company has a long way to go to prove this therapy is truly useful. It spends just 14.4% of its revenue on research, development and clinical trials, just slightly more than what it earned in profits last year.
Now that its drug has clear sailing once again to treat the small patient population that suffers terribly from this disease, surely it can afford to spend the money needed to learn whether it truly works. Commissioner Makary could order Sarepta to create a registry that collects data on every patient that gets treated, which would enable statisticians, regulators and families to track these young patients’ progress and outcomes over time. He could demand Sarepta finally complete a well-controlled clinical trial with endpoints that matter.
In other words, he could require the company meet the evidentiary standards that once earned the FDA the reputation of being the gold standard for regulatory science. Isn’t that more important than keeping one’s job?
(An earlier version of this story incorrectly reported the uses of Elevidys. It has been approved by the FDA for all indications of Duchenne Muscular Dystrophy.)
Merrill Goozner is a former editor of Modern Healthcare, where he wrote a weekly column. He is also a former reporter for The Chicago Tribune and professor of business journalism at New York University.
Reprinted with permission from Gooznews Substack. Please visit and consider subscribing.
