Kresladi (marnetegragene autotemcel) received FDA approval as the first gene therapy for severe Leukocyte Adhesion Deficiency Type I (LAD-I) and is indicated for pediatric patients with LAD-I due to biallelic ITGB2 variants who lack an HLA-matched sibling donor for allogeneic HSCT. The therapy uses the patient’s own hematopoietic stem cells, genetically modified to introduce functional ITGB2, followed by a single intravenous infusion after conditioning, with the aim of restoring CD18 and CD11a surface expression on neutrophils.
Approval relied on a single open-label, multicenter study, measuring increases in neutrophil CD18 and CD11a as disease-specific biomarkers surrogate for clinical benefit, with sustained effects through month 24; post-marketing requirements will confirm clinical benefit. Common adverse events in the study included hematologic declines (anemia, thrombocytopenia, leukopenia), mucosal ulcers, infections, fever, neutropenia, gastrointestinal symptoms, rash, device-related infections, and elevated liver enzymes.
The approval was granted under accelerated pathways with post-approval studies and various designations, including orphan and rare pediatric disease. Uncertainty remains regarding long-term clinical benefit beyond surrogate endpoints pending confirmatory data.
FDA News Releases published a clinical update in Research Highlights on 26 Mar 2026.
The item focuses on FDA Approves First Gene Therapy for Severe Leukocyte Adhesion Deficiency Type I.
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