Rocket says FDA accepts BLA with priority review for RP-L201 to treat LAD-I

Rocket Pharmaceuticals announced that the U.S. Food and Drug Administration or FDA has accepted the Biologics License Application,BLA, and granted Priority Review for RP-L201, marnetegragene autotemcel , a lentiviral vector LV -based investigational gene therapy for severe Leukocyte Adhesion Deficiency-I LAD-I , a rare genetic immune disorder that predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant. The PDUFA date set by the FDA is March 31, 2024. “Today’s acceptance of the BLA by the FDA marks a significant milestone for Rocket towards our goal of delivering a one-time gene therapy to patients facing the devastating effects of severe LAD-I. For these patients, survival beyond childhood is uncommon. Bone marrow transplant is currently the only treatment option, has substantial morbidity and mortality, and may not be available in time for these children,” said Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer, Rocket Pharma. “We are incredibly grateful to the patients, caregivers and researchers who have shared this journey with us and look forward to continuing our close collaboration with the FDA during the review period as we work to bring RP-L201 to patients as quickly as possible.”