What is the story about?
What's Happening?
The FDA has accepted Rocket Pharmaceuticals' resubmission of a biologics license application (BLA) for marnetegragene autotemcel, a gene therapy for leukocyte adhesion deficiency type 1 (LAD-I). The BLA is supported by data from a phase 1/2 clinical trial showing a 100% overall survival rate in patients for at least one year post-treatment. The FDA has set the Prescription Drug User Fee Act (PDUFA) action date for March 28, 2026.
Why It's Important?
The resubmission of Rocket's BLA for LAD-I gene therapy marks a critical step towards potentially offering a new treatment option for patients with this rare genetic disorder. If approved, the therapy could provide a one-time solution for LAD-I, addressing a significant unmet need and improving patient outcomes. The FDA's review process will determine the therapy's availability and impact on the healthcare landscape.
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