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Rocket Pharmaceuticals Announces FDA Approval of KRESLADI™ for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)
First FDA-approved gene therapy for children with severe LAD-I due to biallelic variants in ITGB2 Severe LAD-I is an ultra-rare, life-threatening pediatric
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\nFirst FDA-approved gene therapy for children with severe LAD-I due to biallelic variants in ITGB2\n\n\nSevere LAD-I is an ultra-rare, life-threatening pediatric genetic immunodeficiency characterized by recurrent infections and high early-childhood mortality without treatment\n\n\nFDA grants Rare Pediatric Disease Priority Review Voucher\n\n\nCompany to host conference call today, March 27 at 8:30 AM ET\n\n\n CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for KRESLADI™ (marnetegragene autotemcel), 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. 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.\n\n\nWith the approval of KRESLADI, the FDA granted Rocket a Rare Pediatric Disease Priority Review Voucher (PRV), 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.\n\n\n“The approval of KRESLADI represents an important milestone for the severe LAD-I community,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. “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 United States.”\n\n\nLAD-I is an ultra-rare genetic pediatric disease caused by mutations in the ITGB2 gene encoding for CD18, a key protein that is expressed along CD11 integrins to facilitate leukocyte adhesion t...