Shares of Rocket Pharmaceuticals (NASDAQ:RCKT) rose 6% on Friday after the U.S. Food and Drug Administration granted accelerated approval to KRESLADI, the company’s gene therapy intended for children with severe leukocyte adhesion deficiency-I (LAD-I).
KRESLADI is an autologous hematopoietic stem cell-based gene therapy indicated for pediatric patients with severe LAD-I caused by biallelic variants in the ITGB2 gene, where there is no available human leukocyte antigen (HLA)-matched sibling donor for an allogeneic hematopoietic stem cell transplant. The approval marks the first FDA-sanctioned gene therapy for children diagnosed with the severe form of this condition.
The accelerated approval was granted on the basis of demonstrated increases in neutrophil CD18 and CD11a surface expression. Regulators specified that confirmation of clinical benefit will rely on longer-term follow-up data from the ongoing clinical study and outcomes recorded in a post-marketing registry.
Severe LAD-I is described as an ultra-rare, life-threatening pediatric genetic immunodeficiency resulting from mutations in ITGB2. Patients with the disorder experience recurrent infections and face high mortality in early childhood if left untreated. In the United States, the condition’s incidence is estimated to be between approximately one in 100,000 and one in 200,000 live births, and roughly two-thirds of those affected are classified as having the severe form of LAD-I.
Alongside the approval, the FDA awarded Rocket Pharmaceuticals a Rare Pediatric Disease Priority Review Voucher. The company announced its intent to evaluate strategic options to monetize that voucher as a means to bolster financial flexibility and to pursue actions aimed at maximizing shareholder value.
Gaurav Shah, Chief Executive Officer of Rocket Pharmaceuticals, said of the decision: "The approval of KRESLADI represents an important milestone for the severe LAD-I community."
Research that supported the development of KRESLADI received partial funding from the California Institute for Regenerative Medicine.
Market and sector context
The FDA’s accelerated approval and the issuance of a priority review voucher are developments with implications for the biotechnology and healthcare investment community, particularly companies focused on gene therapies and rare pediatric conditions. The voucher presents a potential near-term financial asset for Rocket if it chooses to monetize it; however, the long-term commercial profile of KRESLADI will depend on confirmatory clinical outcome data.