Dr. Makary and Mr. Hyde at the FDA

The agency kills a therapy for melanoma despite the evidence of progress against deadly tumors

WSJ editorial. Excerpts:

"Patients with metastatic melanoma who stop responding to other immunotherapies typically die in less than a year. In Replimune’s trial, tumors shrank in nearly all patients and vanished in one of six. About a third went into remission. FDA staff were so impressed by the results that the agency designated RP1 a “breakthrough therapy” in November 2024 to expedite its review."

"As we’ve reported, Dr. Prasad last summer overruled career staff to reject RP1. The agency’s main criticism was that its trial lacked a control arm, though this would be unethical in late-stage patients who failed to improve on other therapies."

"Start with the claim that the tumor-shrinking effects of RP1 could not be disentangled from that of another immunotherapy that patients were taking concurrently. But all patients had previously relapsed or failed to respond to other immunotherapies. RP1 is intended to help these refractory patients by boosting their response to other therapies."

"cancer in responding patients advanced after a median 30.6 months when they also got RP1, versus 4.4 months of being treated with other immunotherapies."

"The FDA implicitly concedes that the RP1 results are impressive by contriving ridiculous reasons to argue they could be exaggerated."