Rocket Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to KRESLADI™ (marnetegragene autotemcel), an autologous hematopoietic stem cell–based gene therapy. The therapy is indicated for pediatric patients with severe leukocyte adhesion deficiency type 1 (LAD-I) caused by biallelic variants in the ITGB2 gene who do not have an HLA-matched sibling donor for allogeneic stem cell transplantation.
As per the Rocket Pharmaceuticals press release, the approval is based on the therapy’s ability to increase neutrophil CD18 and CD11a surface expression, markers associated with improved immune function. However, Rocket will confirm the long-term clinical benefit through ongoing patient follow-up in current clinical studies and a post-marketing registry.
Rare Pediatric Disease Voucher Granted
Alongside the approval, the FDA awarded Rocket Pharmaceuticals a Rare Pediatric Disease Priority Review Voucher (PRV). This program encourages the development of treatments for rare pediatric conditions.
Rocket stated that it will evaluate strategic options to monetize the voucher, which could enhance the company’s financial flexibility and shareholder value.
Addressing an Ultra-Rare and Life-Threatening Disorder
Severe LAD-I is an ultra-rare genetic immune disorder caused by mutations in the ITGB2 gene, which encodes the CD18 protein. This protein works alongside CD11 integrins to help white blood cells adhere to blood vessel walls and migrate to infected tissues.
When CD18 function is impaired, the immune system struggles to control infections and support wound healing. As a result, infants with severe LAD-I experience recurrent, life-threatening bacterial and fungal infections, often requiring frequent hospitalization.
In the United States, the condition affects approximately one in 100,000 to one in 200,000 live births, with nearly two-thirds of cases classified as severe.
Experts Highlight a Major Therapeutic Milestone
Gaurav Shah, MD, Chief Executive Officer of Rocket Pharmaceuticals, described the approval as an important milestone for the severe LAD-I community. He emphasized that the achievement reflects the collective efforts of patients, families, researchers, and regulators working to advance treatment for this ultra-rare disease.
Clinical experts also welcomed the development. Donald B. Kohn, MD, principal investigator of the Phase 1/2 study at the University of California, Los Angeles (UCLA), noted that KRESLADI represents years of scientific research and clinical collaboration aimed at addressing the root cause of the disease.
Similarly, Vanessa Tenembaum, CEO of the Jeffrey Modell Foundation, highlighted that the approval marks significant progress for patients with severe LAD-I and the broader primary immunodeficiency community, offering new hope for families affected by this devastating condition.