- Size:
- —
- Delta:
- No change (90.2% → 90.2%)
High YES price reflects strong approval expectations for resubmitted BLA. No cash available to trade.
High YES price reflects strong approval expectations for resubmitted BLA. No cash available to trade.
FDA accepted BLA resubmission in Oct 2025 with March 28, 2026 PDUFA. Previous CRL cited only "limited CMC" issues, likely resolved.
The prior FDA rejection was for manufacturing (CMC) issues, not safety or efficacy. The company has since resubmitted, and with strong 100% survival data, approval odds are very high and priced in.
Resubmitted BLA for orphan gene therapy in severe LAD-I has high approval odds post-CRL fixes and unmet need; approval surges RCKT valuation. Steady-state cap reduced request to $1000.00.
Prior CRL was CMC-driven; even with the resubmitted BLA accepted, another manufacturing/inspection snag can still trigger a CRL. At 90% YES, upside is small vs big down/stock hit if rejected. Steady-state cap reduced request to $1000.00.