FDA accepts BLA and grants Priority Review for Rocket's RP-L201 gene therapy for severe LAD-I; PDUFA March 31, 2024

ROCKET PHARMACEUTICALS, INC.

PDUFA target action date set for March 31, 2024.
RP-L201 has FDA RMAT, Rare Pediatric, Fast Track, and Orphan Drug designations.
Rocket eligible for Priority Review Voucher if RP-L201 is approved.
Phase 1/2 data: 100% overall survival at ≥12 months for all nine patients; symptoms resolved.
RP-L201 is a lentiviral vector-based gene therapy for severe LAD-I, a fatal childhood immune disorder.

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