The US Food and Drug Administration (FDA) has approved Rocket Pharmaceuticals’ Kresladi (marnetegragene autotemcel) in leukocyte adhesion deficiency type I (LAD-I), becoming the first pharmaceutical option for children with the ultra-rare immune disease.
As per the accelerated approval, Kresladi – a gene therapy – is indicated for the treatment of paediatric patients with severe LAD-I due to biallelic variants in ITGB2. Patients eligible for the therapy are those who do not have a matched sibling donor for a stem cell transplant, the current standard of care (SoC) for the disease.
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Severe LAD-I is an inherited immune deficiency caused by mutations in the ITGB2 gene, which prevent white blood cells from effectively fighting infections. The disease leaves children vulnerable to pathogens, with patients incurring recurrent bacterial and fungal infections. LAD-1 is ultra-rare, affecting one in one million individuals.
Kresladi consists of the patient’s own hematopoietic stem cells, which are genetically modified to introduce functional copies of the ITGB2 gene. A single dose of Kresladi is infused intravenously to restore proteins encoded by the gene, which help white blood cells adhere to vessel walls and move to sites of infection.
The FDA based its decision on data from a Phase I/II trial (NCT03812263), which showed the gene therapy increased neutrophil CD18 and CD11a cell surface expression, which are disease-specific biomarkers indicative of improved immune activity. Levels rose at month 12 with sustained effect through month 24 post-infusion. The clinical benefit of Kresladi will be confirmed in patients with severe LAD-I through post-marketing requirements as set out by the FDA.
“The FDA continues to exercise significant regulatory flexibilities as applicable, during both chemistry, manufacturing and control and clinical review of licensing applications. For rare diseases, the FDA considers small patient populations in clinical trials and all available sources of evidence to advance life-changing treatments while still meeting its rigorous scientific standards,” said the FDA’s Center for Biologics Evaluation and Research (CBER) director Vinay Prasad.
Kresladi’s approval might mark the final biologic greenlit by Prasad, who is leaving the agency in April. Prasad will return to his role at the University of California, San Francisco, where he is a professor of epidemiology and biostatistics and medicine. During his time as CBER head, he attracted criticism for his conservative approach to cell and gene therapy approvals.
Given its rarity, Kresladi will likely not be a major revenue driver for Rocket. Global sales for the therapy are predicted to reach $120m in 2031 as per analyst consensus forecasts calculated by GlobalData, parent company of Pharmaceutical Technology.
It will, however, have significant impact on the patient community, said Vanessa Tenembaum, CEO of the Jeffrey Modell Foundation, a global nonprofit organisation dedicated to early diagnosis and treatments for primary immunodeficiency.
“The approval of KRESLADI represents a significant development for individuals affected by severe LAD-I and the broader primary immunodeficiency community. For families impacted by this rare and serious disease this approval underscores the importance of continued efforts to improve outcomes for patients with primary immunodeficiencies,” Tenembaum explained.
Because Kresladi had rare paediatric disease designation, Rocket has been handed a priority review voucher upon the product’s approval. These vouchers can be sold to fellow pharma companies, with a current market price of around $200m. Jazz Pharmaceuticals announced it sold one at this price at the JP Morgan Healthcare Conference in January 2026.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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