The US Food and Drug Administration (FDA) has granted accelerated approval to Kresladi (marnetegragene autotemcel) for the treatment of pediatric patients with severe leukocyte adhesion deficiency-1 (LAD-1). The indication is for patients with biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant.
LAD-1 is a genetic immunodeficiency characterized by serious bacterial and fungal infections. One of the first signs is a delay in the detachment of the umbilical cord stump after birth and inflammation of the umbilical cord stump (omphalitis) due to a bacterial infection. Infections in these patients most commonly occur on the skin and mucous membranes, but they can occur elsewhere. Infants with severe forms of the disease often have recurrent, life-threatening infections that respond poorly to standard therapy. Life expectancy in individuals with LAD-1 is often severely shortened.
Marnetegragene autotemcel is an autologous hematopoietic stem cell-based gene therapy designed to deliver a functional copy of the ITGB2 gene, which encodes for the beta-2 integrin component CD18, a key protein that facilitates leukocyte adhesion and enables their extravasation from blood vessels to fight infection.
The accelerated approval is largely based on data from a phase 1/2 clinical trial showing increased neutrophil CD18 and CD11a surface expression. That study was published last year in the New England Journal of Medicine. Full approval of the gene therapy will require confirmation of clinical benefit based on longer-term follow-up data of treated patients in the ongoing clinical study and through a post-marketing registry.
With the approval, the FDA granted Rocket Pharmaceuticals a Rare Pediatric Disease Priority Review Voucher (PRV), a program designed to encourage development of therapies for rare pediatric diseases.
For more information, visit https://www.businesswire.com/news/home/20260326279809/en/Rocket-Pharmaceuticals-Announces-FDA-Approval-of-KRESLADI-for-Pediatric-Patients-with-Severe-Leukocyte-Adhesion-Deficiency-I-LAD-I
To learn more about LAD-1 and other rare autoimmune conditions, visit https://checkrare.com/diseases/autoimmune-autoinflammatory-disorders/
