FDA Approves Gene Therapy for Severe Pediatric LAD-I
Approval expands treatment option for a rare pediatric immunodeficiency
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By
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Kathryn Wighton
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March 27, 2026
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Clinical Scorecard: FDA Approves Gene Therapy for Severe Pediatric LAD-I
At a Glance
| Category | Detail |
|---|
| Condition | Severe Leukocyte Adhesion Deficiency Type I (LAD-I) |
| Key Mechanisms | Caused by biallelic variants in ITGB2 affecting CD18 protein function in leukocyte adhesion. |
| Target Population | Pediatric patients without an available human leukocyte antigen-matched sibling donor. |
| Care Setting | Clinical settings for gene therapy and hematopoietic stem cell transplant. |
Key Highlights
- Accelerated approval of marnetegragene autotemcel (KRESLADI) by the FDA.
- Indicated for severe LAD-I with no matched sibling donor.
- Condition associated with high mortality in early childhood without treatment.
- Observed increases in neutrophil CD18 and CD11a surface expression post-treatment.
- Continued approval contingent on long-term clinical benefit verification.
Guideline-Based Recommendations
Diagnosis
- Diagnosis based on genetic testing for biallelic variants in ITGB2.
Management
- Consider marnetegragene autotemcel for eligible pediatric patients.
Monitoring & Follow-up
- Long-term follow-up required for clinical benefit verification and monitoring of safety.
Risks
- Risks include serious infections, veno-occlusive disease, and potential oncogenesis.
Patient & Prescribing Data
Pediatric patients with severe LAD-I and no matched sibling donor.
Autologous hematopoietic stem cell-based gene therapy.
Clinical Best Practices
- Conduct thorough genetic testing for ITGB2 variants.
- Monitor for potential adverse effects post-gene therapy.
- Engage in multidisciplinary care for management of severe LAD-I.
References
