FDA Clears First Gene Therapy for Rare Pediatric Immune Disorder

The U.S. Food and Drug Administration (FDA) has approved Kresladi, the first gene therapy for severe Leukocyte Adhesion Deficiency Type I (LAD-I), a rare and life-threatening genetic condition affecting the immune system.

The therapy is indicated for pediatric patients with mutations in the ITGB2 gene who do not have a suitable donor for stem cell transplantation. LAD-I prevents white blood cells from effectively fighting infections, leading to recurrent and often fatal bacterial and fungal complications early in life.

Kresladi is an autologous treatment that uses the patient’s own hematopoietic stem cells, genetically modified to correct the underlying defect. Once reinfused, these cells restore key immune functions by enabling proper expression of proteins essential for leukocyte activity.

The approval was granted under an accelerated pathway, based on clinical data showing sustained improvements in immune biomarkers, including CD18 and CD11a expression, for up to two years after treatment. These markers are considered predictive of clinical benefit.

This approval is significant because it represents the first FDA-authorized gene therapy for a severe pediatric immunodeficiency, offering a potentially life-saving treatment for children who previously had no effective options and highlighting a major step forward in precision medicine for rare diseases.

SOURCE: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-severe-leukocyte-adhesion-deficiency-type-i 

CREDITS: U.S. FOOD AND DRUG ADMINISTRATION