Rocket Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for its gene therapy KRESLADI™ for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I). With the approval, the FDA granted the company a Rare Pediatric Disease Priority Review Voucher, which it intends to evaluate strategic options to monetize.
Key takeaways
FDA granted accelerated approval for KRESLADI™ (marnetegragene autotemcel) gene therapy on March 27, 2026.
KRESLADI™ is indicated for pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available HLA-matched sibling donor.
Approval was based on increase in neutrophil CD18 and CD11a surface expression, with confirmation of clinical benefit pending longer-term follow-up data.
FDA granted Rocket Pharmaceuticals a Rare Pediatric Disease Priority Review Voucher (PRV) with the approval.
Company intends to evaluate strategic options to monetize the PRV to enhance financial flexibility and maximize shareholder value.
Company will host an investor conference call on March 27, 2026, at 8:30 AM ET.