Posted by Michael Wonder on 03 Oct 2023
Rocket Pharmaceuticals announces FDA acceptance of biologics license application with priority review for RP-L201 (marnetegragene autotemcel) for the treatment of severe leukocyte adhesion deficiency-I
2 October 2023 - PDUFA target action date is 31 March 2024.
Rocket Pharmaceuticals today announced that the US FDA has accepted the biologics license application and granted priority review for RP-L201 (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.
