Exclusive | How Replimune’s Drug Got Third Chance After White House Intervention - WSJ

Replimune, the clinical-stage biotech firm, is getting a third shot at FDA approval for its lead oncolytic immunotherapy, RP1, following what sources describe as a "White House intervention" and a significant shake-up at the agency. This unprecedented move, weeks after the departure of influential FDA advisor Dr. Marty Makary, has sent Replimune stock soaring and ignited fierce debate over regulatory independence versus presidential pressure on drug access. The FDA had twice rejected RP1 for metastatic melanoma and cutaneous squamous cell carcinoma, citing insufficient efficacy data and concerns about its benefit-risk profile, especially when used in combination with PD-1 inhibitors like nivolumab. Makary, known for his critical stance on accelerated approvals without robust long-term data, was reportedly a strong voice internally against the drug. His exit appears to have cleared a path for the Biden administration to push for a re-evaluation, possibly influenced by a broader policy directive to streamline novel cancer drug access amid growing public demand for therapeutic options. Replimune is now preparing a revised Biologics License Application (BLA), likely leveraging new analyses or a more defined patient sub-group under the Accelerated Approval Pathway. This development sets a potentially controversial precedent for how political influence might shape scientific regulatory decisions, forcing the BioPharma sector to weigh the potential for faster market access against the perception of a compromised FDA. All eyes will be on the agency's review, scrutinizing whether scientific rigor ultimately prevails or if policy imperatives take the lead.