On Tuesday, the U.S. Food and Drug Administration (FDA) accepted Rocket Pharmaceuticals, Inc.’s (NASDAQ:RCKT) resubmission of the Biologics License Application (BLA) for Kresladi (marnetegragene autotemcel; marne-cel), a lentiviral vector (LV)-based investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I).
LAD-I is a rare genetic immune disorder that predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant.
The PDUFA date set by the FDA is March 28, 2026.
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The BLA is supported by positive clinical efficacy and safety data from the global Phase 1/2 study of Kresladi, which demonstrated 100% overall survival at 12 months post-infusion (and for the entire duration of follow-up) for all enrolled patients.
All primary and secondary endpoints were met, and Kresladi was well tolerated in all patients with no treatment-related serious adverse events. The data showed substantial reductions in the incidence of significant infections compared to pre-treatment levels, along with evidence of improvement in skin lesions and restoration of wound-healing capabilities.
Rocket is eligible for a Rare Pediatric Disease Priority Review Voucher (PRV) should Kresladi be approved.
William Blair on Tuesday wrote, “We view the Kresladi acceptance positively as it reestablishes momentum for a program that had previously secured priority review and has demonstrated compelling durability. With the prior CMC concerns addressed, we remain optimistic…”
Analyst Sami Corwin anticipates a potential launch in the third quarter of 2026, modeling peak sales of $294 million in 2031.
“However, we recognize that the company has stated that it plans to focus its resources more heavily on its late-stage AAV pipeline, suggesting commercialization efforts for Kresladi could be modest,” Corwin wrote on Tuesday.
Price Action: RCKT stock was up 18.01% at $4.09 at the last check on Tuesday.
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