By Ahmed Aboulenein and Puyaan Singh
WASHINGTON, April 22 (Reuters) - Health Secretary Robert F. Kennedy Jr. told a Senate hearing on Wednesday that he had nothing to do with the U.S. FDA's decision to not approve Replimune's drug for advanced skin cancer, saying it was in the hands of the agency's Commissioner Dr. Marty Makary.
Kennedy, at the hearing to discuss President Donald Trump's budget for the Department of Health and Human Services for fiscal 2027, said he "had nothing to do with this decision."
Earlier this month, the Food and Drug Administration declined to approve Replimune's drug, RP1, taking issue with the company's reliance on a single‑arm study for the medicine without a control group. In its rejection letter, the agency said the company must provide data from a well-controlled trial demonstrating adequate evidence of effectiveness.
"This decision comes out of FDA, and we trust the process there. And I've been told by Marty Makary that every panel that looked at that drug unanimously voted against it... because it does not appear to work," Kennedy said.
Replimune shares have fallen nearly 70% since the FDA's rejection of the drug for the second time in two years.
However, shares were up 15% on Wednesday following an opinion piece in The Wall Street Journal late on Tuesday that said Kennedy's comments were not true.
It cast blame on the FDA's drug head Vinay Prasad, who has come under fire before in the Journal for his regulatory decisions. Prasad said last month he would be leaving the agency in April.
The opinion piece cited cancer doctors who have worked on trials of the drug, saying it was effective. The paper called out Kennedy for saying at a Capitol Hill hearing last week that Makary "made the correct decision to not approve that drug."
It said "denying patients a life-saving medicine for inexplicable reasons is the wrong kind of change. If Dr. Makary doesn’t understand that, the FDA needs a change in leadership."
Replimune, following the April 10 rejection, said it disagreed with the FDA about whether the data set was sufficient for approval, adding that the agency appeared to have contradicted its positions expressed at a meeting in September.
Replimune did not immediately respond to a request for comment.
Andrew Nixon, spokesperson for HHS which oversees the FDA, said "FDA career scientists and senior office leadership in the Center for Biologics Evaluation and Research unanimously determined that the current evidence for (Replimune's drug) does not meet the evidentiary standards required for regulatory approval."
(Reporting by Puyaan Singh in Bengaluru and Ahmed Aboulenein in Washington; Editing by Caroline Humer and Bill Berkrot)
