Biotechnology company Rocket Pharmaceuticals Inc (NASDAQ:RCKT) announced on Friday that the US Food and Drug Administration (FDA) has granted accelerated approval for Kresladi (marnetegragene autotemcel) for paediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) without an available human leukocyte antigen-matched sibling donor.

The therapy represents the first FDA-approved gene therapy for children with severe LAD-I caused by biallelic variants in ITGB2.

This approval is supported by increases in neutrophil CD18 and CD11a surface expression, with confirmation of clinical benefit expected from longer-term follow-up data and a post-marketing registry.

LAD-I is an ultra-rare, life-threatening genetic immunodeficiency characterised by recurrent infections and high early-childhood mortality without treatment, with severe cases marked by very low CD11a expression and frequent hospitalisations.

Alongside the approval, Rocket Pharmaceuticals received a Rare Pediatric Disease Priority Review Voucher, which the company plans to evaluate for monetisation to support financial flexibility and shareholder value.