FDA approves Rocket Pharmaceuticals’ Kresladi in LAD-I

According to the post on the FDA website, the agency “approved Kresladi, marnetegragene autotemcel, the first gene therapy for the treatment of severe Leukocyte Adhesion Deficiency Type I, or LAD-I. Kresladi is indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency I due to biallelic variants in ITGB2 without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant… The FDA granted accelerated approval of Kresladi, as well as a Rare Pediatric Disease Priority Review Voucher, to Rocket Pharmaceuticals (RCKT), Inc. As a condition of accelerated approval, Rocket Pharmaceuticals, Inc., is required to conduct post-approval studies to verify and describe the clinical benefit of Kresladi. Continued approval may be contingent upon verification of clinical benefit in confirmatory trials.”