Rocket Pharmaceuticals Announces FDA Acceptance of BLA Resubmission for KRESLADI™ Gene Therapy for Severe LAD-I; PDUFA Date Set for March 2026
Rocket Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the resubmission of the Biologics License Application (BLA) for KRESLADI™ (marnetegragene autotemcel; marne-cel), an investigational gene therapy for the treatment of severe Leukocyte Adhesion Deficiency-I (LAD-I). The FDA has set a Prescription Drug User Fee Act (PDUFA) date of March 28, 2026, for its decision. This BLA resubmission is supported by positive clinical data showing 100% overall survival at 12 months post-infusion among treated patients. If approved, Rocket Pharmaceuticals may be eligible for a Rare Pediatric Disease Priority Review Voucher. No grant or funding announcement involving multiple organizations was disclosed.
Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Rocket Pharmaceuticals Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 20251013059069) on October 14, 2025, and is solely responsible for the information contained therein.
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