Rocket Pharmaceuticals (NASDAQ:RCKT), a leading biotechnology firm specializing in genetic therapies for rare disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for KRESLADI™ (marnetegragene autotemcel).
The therapy is indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) caused by mutations in the ITGB2 gene who lack a matched sibling donor for a stem cell transplant.
KRESLADI™ is an autologous, gene-modified hematopoietic stem cell therapy designed to restore the expression of CD18, a critical protein that allows white blood cells to adhere to blood vessel walls and migrate to sites of infection.
Children with severe LAD-I suffer from recurrent, life-threatening infections and poor wound healing, often failing to survive past infancy without successful intervention.
The FDA’s decision was based on clinical data showing a significant increase in neutrophil CD18 and CD11a surface expression, with long-term clinical benefit to be confirmed via a post-marketing registry.
In conjunction with the approval, the FDA awarded Rocket a Rare Pediatric Disease Priority Review Voucher (PRV).
CEO Gaurav Shah, M.D., indicated that the company intends to explore strategic options to monetize this voucher, which historically commands a market value between $95 million and $110 million.
This potential non-dilutive capital would significantly enhance Rocket's financial flexibility as it advances its late-stage pipeline, including therapies for Danon Disease and Fanconi Anemia.