Facing increasing pressure from both industry and the White House, FDA Commissioner Marty Makary said the strong bad press against him is “corporate spin” and that the agency has “followed the science.”
FDA Commissioner Marty Makary has hit back anew at criticism that he and his agency have received in recent months over contentious drug decisions and mounting internal disarray.
“I stand by the scientists at FDA,” Makary said in an interview with CNBC on Tuesday. “On my watch, we have not done corrupt sweetheart deals. What we have done is followed the science.”
Much of the blowback against Makary, according to CNBC, stems from the agency’s controversial rejection of Replimune’s melanoma therapy RP1. The denial was due largely to Replimune’s insistence of using a single-arm trial to back its application, according to the FDA’s Complete Response Letter, which was made publicly available in line with the agency’s effort last July to release all rejection letters.
RP1 had already been rejected before, in July 2025. The FDA at the time similarly raised issues with Replimune’s use of a single-arm study.
“The FDA clearly recommended that control group” to Replimune, Makary said on the CNBC interview, but “the company clearly did not do that control group.”
Analysts at BMO Capital Markets seem to agree with Makary on this point. “Commentary from the CRL clarify the consistency in FDA’s decision to deny approval,” the firm wrote in an April 10 note. The letter makes clear that the company and the regulator had never truly aligned on a program design—even though Replimune had previously suggested that they had.
“While the FDA allowed the resubmission, they appeared to do so out of a desire to be open to the possibility that fundamental issues with the product’s clinical package could be addressed in a high unmet need indication,” BMO added.
Makary called the strong pushback in the press against the Replimune rejection “corporate spin,” singling out the “nine articles on one drug” from The Wall Street Journal. “The full story is in our complete response letter that we published.”
Aside from the Replimune rejection, the FDA also faced controversy late last year when uniQure was informed that a Phase 1/2 program would no longer be enough to support an application for a Huntington’s gene therapy—a “key shift” from the agency’s previous guidance, the company said at the time. UniQure has raised concerns that a sham-controlled study, which would satisfy the FDA’s evidentiary requirements, would come with steep ethical issues.
But Makary insisted that all decisions have been made in accordance with the findings of review teams. “Every accept or reject decision at the FDA, on my watch, has been the accept or reject recommendation of the primary review teams at the agency,” he insisted. “I’ve spent my whole career taking care of cancer patients. To suggest that we want cancer patients to die is a little offensive.”
In addition to pushback on the FDA’s review decisions, pressure on Makary also seems to be building from inside the agency. Sources at the FDA say that one more big mistake could endanger Makary’s job, according to reporting from Bloomberg. There are currently no plans to replace him, the insiders clarified, but the situation is tenuous.
The frustration over Makary’s performance has also reached the inner circles of President Donald Trump. Earlier this year, after the FDA refused to even review Moderna’s mRNA flu vaccine, the commissioner was summoned to the White House, after which the agency and Moderna reached a compromise. The FDA has now accepted the filing for review, with a decision due in August.
The discontent over Makary has only grown since, NOTUS reported on Tuesday, citing two anonymous sources. Administration officials have grown weary over Makary’s failure to “wrangle the bureaucracy and move quickly,” according to one of the sources, who added that the commissioner hasn’t been “responsive enough” to other concerns regarding the FDA.
In a statement to NOTUS, a spokesperson for the White House maintained that Makary “continues to be an invaluable asset for the Trump administration.”
