Story perspectives

FDA Advances Rocket's KRESLADI for Rare Blood Disorder Treatment

10/15/2025

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Summary

Story summary

The U.S. FDA accepted Rocket Pharmaceuticals, Inc.'s resubmission of the Biologics License Application for KRESLADI (marnetegragene autotemcel) to treat LAD-I.
The target action date is March 28, 2026.
Clinical data show 100% overall survival at 12 months post-infusion, with reduced infections and improved wound healing.
Rocket is eligible for a Rare Pediatric Disease Priority Review Voucher if approved.
Analysts expect a potential late-2026 launch, despite focusing on other late-stage therapies.

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Story summary

The U.S. FDA accepted Rocket Pharmaceuticals, Inc.'s resubmission of the Biologics License Application for KRESLADI (marnetegragene autotemcel) to treat LAD-I.
The target action date is March 28, 2026.
Clinical data show 100% overall survival at 12 months post-infusion, with reduced infections and improved wound healing.
Rocket is eligible for a Rare Pediatric Disease Priority Review Voucher if approved.
Analysts expect a potential late-2026 launch, despite focusing on other late-stage therapies.