FDA Approves Rocket Pharma's Kresladi Gene Therapy for Ultra-Rare LAD-I After Setbacks

The FDA granted accelerated approval to Rocket Pharmaceuticals' Kresladi (marnetegragene autotemcel) on March 26, 2026, for pediatric patients with severe leukocyte adhesion deficiency type I (LAD-I) due to biallelic ITGB2 variants, without a matched sibling donor for stem cell transplant.1 2 4 6

LAD-I is an ultra-rare immune disorder affecting about 1 in 1 million people, causing recurrent life-threatening infections and high childhood mortality without treatment.1 2 4

The therapy was initially rejected by the FDA in 2024 due to manufacturing concerns but approved after resubmission, with PDUFA date of March 28, 2026.1 2 5

Approval is based on Phase I/II trial data showing increased neutrophil CD18 and CD11a expression sustained through 24 months; clinical benefits to be confirmed post-marketing.4 6

Rocket received a Rare Pediatric Disease Priority Review Voucher and expects limited revenue (e.g., $120M by 2031) due to rarity (~25 new US cases/year), but it's a milestone for patients.1 3 4 6