Rocket Pharmaceuticals has received FDA accelerated approval for KRESLADI (marnetegragene autotemcel), an autologous hematopoietic stem cell-based gene therapy for pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 who lack a human leukocyte antigen-matched sibling donor for allogeneic stem cell transplant. The product is the first FDA-approved gene therapy for children with severe LAD-I and is approved based on increased neutrophil CD18 and CD11a surface expression, with continued approval contingent on verification of clinical benefit in longer-term follow-up and a post-marketing registry.
Severe LAD-I is an ultra-rare, life-threatening genetic immunodeficiency marked by recurrent, serious bacterial and fungal infections, poor response to antimicrobials, and high early-childhood mortality without treatment. U.S. incidence is estimated at roughly one in 100,000 to one in 200,000 live births, with about two-thirds of patients classified as severe.
With KRESLADI’s approval, FDA granted Rocket a Rare Pediatric Disease Priority Review Voucher, which the company plans to monetize to enhance financial flexibility and shareholder value. Rocket CEO Gaurav Shah, M.D., called the decision an important milestone for the severe LAD-I community and highlighted the dedication of patients, families, investigators, and regulators.
The indication carries extensive safety guidance, including boxed warnings on risks such as serious infections, veno-occlusive disease, neutrophil and platelet engraftment issues, lentiviral insertional oncogenesis, and hypersensitivity reactions.
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