Rocket Pharmaceuticals (NASDAQ: RCKT) has announced FDA approval of KRESLADI, an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant.
According to Rocket, this is the first FDA-approved gene therapy for children with severe LAD-I due to biallelic variants in ITGB2. With this approval, the FDA granted the company a Rare Pediatric Disease Priority Review Voucher. Rocket intends to evaluate strategic options to monetize the voucher to enhance financial flexibility and maximize shareholder value.
In a statement, Gaurav Shah, MD, CEO of Rocket, commented, “The approval of KRESLADI represents an important milestone for the severe LAD-I community. This approval reflects the dedication of patients, families, investigators, and regulators who have worked together to advance research of this ultra-rare disease. We look forward to making KRESLADI available to eligible patients in the U.S.”
