Without naming a specific product, Commissioner Marty Makary referred to an investigational therapy, delivered surgically into the brain, that the FDA was “pressured” to approve even after finding no clinical benefit to patients.
FDA Commissioner Marty Makary is standing by his agency’s drug review decisions amid recent controversial denials.
“There’s always going to be two sides to a story,” Makary said Thursday morning in an interview with CNBC. “There are moneyed interests,” he continued. “I get it, if I were an investor and I put in $100 million into a product and you didn’t get the results that you like—it is disappointing.”
His remarks were a response to criticism that one of his deputies, Center for Biologics Evaluation and Research Director Vinay Prasad, had been coming in late in the review process to change previous agency advice to a drug sponsor—ultimately resulting in a rejection despite the company having followed that advice.
The FDA’s initiative to publicly release complete response letters, Makary continued, is a step toward making the regulator’s rationale behind a rejection as visible as possible, though he conceded that the move has led to “a bit of an effort to find a bogeyman”—a reason that can account for a denial.
For example, “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 said. “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.”
Makary did not name the drug candidate, but his comments seem to match uniQure’s gene therapy AMT-130. AMT-130, a treatment for Huntington’s disease, is delivered directly into the brain via a neurosurgical procedure.
In November 2025, the FDA told uniQure that it “no longer agrees” that a Phase 1/2 program would be enough to support an approval. The biotech called it a “key shift” after the regulator had previously signed off on the study plan for AMT-130.
“Consequently, the timing of the BLA submission for AMT-130 is now unclear,” uniQure said at the time.
The biotech’s shares closed Thursday’s trading session at $17.11 apiece, a 31% crash from its previous closing price of $24.87.
“To be clear, his specific commentary might/might not have been referring to AMT-130 since QURE never received an actual” rejection letter, analysts at Stifel told investors in a Thursday note. “There has been increasing political pressure on the FDA to be more flexible and one has to wonder if FDA leadership will remain the same during this current Trump term.”
More broadly, Stifel noted that Makary’s “steadfast defense” of the FDA’s approach to rare disease could be “discouraging for those hoping that the agency can be convinced to reconsider recent negative decisions, in the absence of specific political pressure.”
The rare disease space has suffered several high-profile rejections in recent months, including Disc Medicine’s bitopertin for erythropoietic protoporphyria, REGENXBIO’’s RGX-121 for Hunter syndrome and Ultragenyx’s UX111 for Sanfilippo syndrome.
