FDA Acceptance Of Rocket Pharmaceuticals' Lead Gene Therapy Application Reestablishes Momentum For Stock

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]