Rocket Pharma: FDA accepts BLA resubmission for KRESLADI gene therapy; PDUFA March 28, 2026

ROCKET PHARMACEUTICALS, INC.

FDA accepted BLA resubmission for KRESLADI (marne-cel) for severe LAD-I; PDUFA target date March 28, 2026.
Phase 1/2 study showed 100% overall survival at 12 months; all endpoints met; well tolerated with no treatment-related SAEs.
Company eligible for Rare Pediatric Disease Priority Review Voucher if KRESLADI is approved.
Severe LAD-I is near-uniformly fatal in childhood; only current treatment is bone marrow transplant with substantial morbidity.

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