Rocket Pharmaceuticals has won FDA accelerated approval for KRESLADI (marnetegragene autotemcel), marking the first gene therapy available for children with severe leukocyte adhesion deficiency-I (LAD-I) caused by biallelic ITGB2 mutations.

 

The approval covers pediatric patients without a matched sibling donor for stem cell transplant. Severe LAD-I is an ultra-rare, life-threatening genetic immunodeficiency that leaves infants vulnerable to recurrent bacterial and fungal infections and carries a high risk of early-childhood mortality.

 

KRESLADI’s approval is based on increased neutrophil CD18 and CD11a expression, with long-term clinical benefits to be confirmed through ongoing studies and a post-marketing registry. In addition, the FDA granted Rocket a Rare Pediatric Disease Priority Review Voucher, which the company plans to explore monetizing to enhance financial flexibility and shareholder value.

 

“The approval of KRESLADI represents an important milestone for the severe LAD-I community,” said Gaurav Shah, Chief Executive Officer of Rocket Pharmaceuticals. “This approval reflects the dedication of patients, families, investigators, and regulators who have worked together to advance research of this ultra-rare disease. We look forward to making KRESLADI available to eligible patients in the United States.”

 

Donald B. Kohn, Principal Investigator of the Phase 1/2 study and Distinguished Professor at UCLA, added: “As a clinician, I have seen firsthand the serious impact that severe LAD-I can have on young children and their families. The approval of KRESLADI represents the culmination of many years of scientific research and clinical collaboration aimed at addressing the underlying cause of this devastating disease.”

 

Vanessa Tenembaum, CEO of the Jeffrey Modell Foundation, said: “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.”