Posted by Michael Wonder on 16 Oct 2025
Rocket Pharmaceuticals announces FDA acceptance of BLA resubmission of Kresladi for the treatment of severe leukocyte adhesion deficiency-I
14 October 2025 - PDUFA target action date is 28 March 2026.
Rocket Pharmaceuticals today announced that the US FDA has accepted the resubmission of the biologics license application for Kresladi (marnetegragene autotemcel), a lentiviral vector-based investigational gene therapy for severe leukocyte adhesion deficiency-I, a rare genetic immune disorder that predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic haematopoietic stem cell transplant.
