Replimune's core argument against the FDA's rejection is that the entire melanoma medical community is demanding access to their drug. The company highlights that 22 trial investigators and major medical institutions wrote letters to the FDA, attempting to use the weight of expert opinion to overrule the regulator's objections.
The FDA advised against a single-arm study unless data was "sufficiently compelling." Replimune argues the FDA's subsequent actions—granting priority review and not objecting to the filing—implied the data met this bar, making the final rejection based on the study design feel disingenuous and like a retrospective goalpost shift.
After the first FDA rejection, Replimune's chairman reveals they lacked funds to complete their confirmatory study to statistical significance. This forced them to submit a weaker "descriptive analysis" of early data for their second review—a high-risk gamble dictated by financial reality rather than an optimal regulatory strategy.
Facing a negative FDA decision, Replimune has filed a Freedom of Information Act (FOIA) request to investigate why senior FDA leadership allegedly overruled its own review team. This legalistic tactic moves beyond scientific debate into a public challenge of the agency's internal processes and transparency.