Core Insights - Rocket Pharmaceuticals (RCKT) has had its resubmitted biologics license application (BLA) for gene therapy Kresladi accepted by the FDA, aiming to treat severe leukocyte adhesion deficiency-I (LAD-I) [1][7] - The FDA's decision on the BLA is anticipated by March 28, 2026, with the potential approval marking RCKT's first commercial product [1][10] - Following the announcement, RCKT's shares increased by 23.1% [1] Company Performance - Year-to-date, RCKT's shares have decreased by 66.1%, contrasting with a 7.7% increase in the industry [4] Clinical Data - The BLA is supported by positive results from a global phase I/II study, demonstrating 100% overall survival for LAD-I patients at 12 months post-infusion, meeting all primary and secondary endpoints [2] - Kresladi treatment was well tolerated, with no serious adverse effects reported [2] Regulatory Incentives - If approved, RCKT will qualify for a Rare Pediatric Disease Priority Review Voucher (PRV), which can expedite the approval of another drug or be sold to a larger pharmaceutical company [3] Background on LAD-I - LAD-I is a rare genetic disorder caused by a defect in the ITGB2 gene, leading to severe immune system issues and recurrent infections [9]

Core Insights - The FDA has accepted Rocket Pharmaceuticals' resubmission of the Biologics License Application for Kresladi, a gene therapy for severe Leukocyte Adhesion Deficiency-I [1][2] - The PDUFA date for Kresladi is set for March 28, 2026 [2] - The BLA is supported by positive clinical data showing 100% overall survival at 12 months post-infusion in a global Phase 1/2 study [3][4] Clinical Data - All primary and secondary endpoints were met in the clinical study, with Kresladi being well tolerated and no serious adverse events reported [4] - Significant reductions in the incidence of infections were observed compared to pre-treatment levels, along with improvements in skin lesions and wound healing [4] Market Outlook - Analyst Sami Corwin from William Blair views the acceptance positively, anticipating a potential launch in Q3 2026 and modeling peak sales of $294 million by 2031 [5] - The company plans to focus more on its late-stage AAV pipeline, which may lead to modest commercialization efforts for Kresladi [6] Stock Performance - Following the news, Rocket Pharmaceuticals' stock increased by 18.01%, reaching $4.09 [6]