Overview
- FDA accelerated approval on Friday authorizes Kresladi for children with severe LAD‑1 caused by biallelic ITGB2 variants who lack a matched sibling donor for stem‑cell transplant.
- Kresladi uses a patient’s own blood stem cells that are engineered with a lentiviral vector to add a working ITGB2 gene, given after myeloablative conditioning such as busulfan.
- Regulators relied on a nine‑patient phase 1/2 study showing sustained increases in neutrophil CD18 and CD11a through 24 months and fewer severe infections, with benefit to be confirmed in ongoing follow‑up and a postmarketing registry.
- Short‑term side effects in the study included anemia, low platelets and white cells, mouth sores, fevers, and infections, and the company warns of a lifelong risk of blood cancers due to insertional oncogenesis from the lentiviral vector.
- Rocket received a Rare Pediatric Disease Priority Review Voucher it plans to sell, its shares rose after the decision, and the approval follows a 2024 FDA rejection over manufacturing issues for a therapy serving an ultra‑rare population often without safe donor options.