The FDA has granted accelerated approval to Kresladi (marnetegragene autotemcel), the first gene therapy for severe leukocyte adhesion deficiency Type 1, a rare pediatric immune disorder.
Kresladi is indicated for pediatric patients with the condition caused by biallelic ITGB2 variants who lack a matched sibling donor for stem cell transplant, according to a March 26 FDA news release. The therapy uses a patient’s own genetically modified hematopoietic stem cells to restore CD18 and CD11a protein expression, targeting the underlying cause of the disease.
The approval was based on a single-arm, multicenter study showing sustained biomarker improvements at 12 months post-infusion, with effects maintained through 24 months. Continued approval is contingent on confirmatory trial data.
The most common side effects included anemia, low blood counts, infections and mouth sores.
The FDA also granted the therapy multiple expedited designations and awarded a Rare Pediatric Disease Priority Review Voucher to Rocket Pharmaceuticals.
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