Rocket Pharmaceuticals announces FDA approval of Kresladi

Rocket Pharmaceuticals (RCKT) announced that the U.S. Food and Drug Administration, FDA, has granted accelerated approval for Kresladi, an autologous hematopoietic stem cell-based gene therapy 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-matched sibling donor for allogeneic hematopoietic stem cell transplant. This indication is approved under accelerated approval based on increase in neutrophil CD18 and CD11a surface expression. Confirmation of clinical benefit will be based on the evaluation of longer-term follow-up data of treated patients in the ongoing clinical study and through a post-marketing registry. With the approval of Kresladi, the FDA granted Rocket a Rare Pediatric Disease Priority Review Voucher, a program designed to encourage development of therapies for rare pediatric diseases. The Company intends to evaluate strategic options to monetize the PRV in a manner designed to enhance financial flexibility and maximize shareholder value.