Immunodeficiency, Leukocyte Adhesion Deficiency
RCKTRocket PharmaceuticalsCatalyst overview
Research summary
This catalyst tracks a Approved update for KRESLADI (Marnetegragene autotemcel) /RP-L201 in Immunodeficiency, Leukocyte Adhesion Deficiency. The expected timing is based on the latest available company guidance and may change.
Q2 2023- BLA submitted. 10/2/23- Accepted BLA. 6/28/24- FDA-issued CRL. 10/14/25- Resubmitted BLA accepted with PDUFA date March 28, 2026. 11/06/25:- Program remains on track for the March 28, 2026 PDUFA action date, and approval could make Rocket eligible for a rare pediatric disease priority review voucher. 02/26/26:- KRESLADI remains on track for March 28, 2026 PDUFA decision for severe LAD-I. 03/27/26:- FDA granted accelerated approval for KRESLADI in severe LAD-I and awarded Rare Pediatric Disease PRV. 05/07/26:- KRESLADI accelerated approval reiterated; associated Rare Pediatric Disease PRV sold for $180 million to strengthen cash runway. 06/12/26:- Closed $180M PRV sale; pro forma cash ~$322.6M; runway expected into Q2 2028. 08/11/26:- KRESLADI commercial availability and patient onboarding expected Q4 2026; PRV sale generated $180M.
Verification
Confirm timing and program details against the company announcement or filing.