Rocket Pharmaceuticals stock gains on FDA gene therapy approval

Rocket Pharmaceuticals (NASDAQ: RCKT) shares rose 6% Friday following the U.S. Food and Drug Administration’s accelerated approval of KRESLADI, the company’s gene therapy for pediatric patients with severe leukocyte adhesion deficiency-I.

KRESLADI, an autologous hematopoietic stem cell-based gene therapy, is indicated for treating pediatric patients with severe LAD-I due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant. The approval marks the first FDA-approved gene therapy for children with severe LAD-I.

The accelerated approval is based on increased neutrophil CD18 and CD11a surface expression. Confirmation of clinical benefit will depend on longer-term follow-up data from the ongoing clinical study and a post-marketing registry.

Severe LAD-I is an ultra-rare, life-threatening pediatric genetic immunodeficiency caused by mutations in the ITGB2 gene. The condition is characterized by recurrent infections and high early-childhood mortality without treatment. In the U.S., the incidence of LAD-I is estimated to range from approximately one in 100,000 to one in 200,000 live births, with roughly two-thirds of affected patients classified as having the severe form.

With the approval, the FDA granted Rocket Pharmaceuticals a Rare Pediatric Disease Priority Review Voucher. The company said it intends to evaluate strategic options to monetize the voucher to enhance financial flexibility and maximize shareholder value.