Rocket Pharmaceuticals Announces FDA Approval of KRESLADI™ for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)
Business Wire • • business
Key Points:
- The FDA has granted accelerated approval to Rocket Pharmaceuticals' KRESLADI™ (marnetegragene autotemcel), a gene therapy for pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) lacking a matched sibling donor for stem cell transplant.
- KRESLADI’s approval is based on increased neutrophil CD18 and CD11a surface expression, with continued approval contingent on confirmatory clinical benefit data from ongoing studies and post-marketing registries.
- The FDA awarded Rocket a Rare Pediatric Disease Priority Review Voucher (PRV), which the company plans to monetize to enhance financial flexibility and shareholder value.
- LAD-I is an ultra-rare genetic disorder causing life-threatening infections in infants due to mutations in ITGB2; KRESLADI offers a new treatment option where conventional therapies are limited.
- Serious risks associated with KRESLADI include infections, veno-occlusive disease, engraftment failures, insertional oncogenesis, and hypersensitivity reactions, necessitating careful patient monitoring and management post-infusion.
