FDA Decision on New RDEB Gene Therapy Expected by April 29, 2025
Exciting News from Abeona Therapeutics — The U.S. Food & Drug Administration (FDA) has accepted Abeona Therapeutics’ resubmitted Biologics License Application (BLA) for Pz-cel (prademagene zamikeracel), a potential new gene therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB). This acceptance means that the FDA is actively reviewing the therapy, with a decision expected by April 29, 2025.
If approved, pz-cel would be the first cell-based gene therapy for RDEB. This therapy is designed to deliver a stable copy of the COL7A1 gene directly to wound sites, helping produce collagen VII, a critical protein that RDEB patients lack. The therapy's effectiveness and safety were studied in a major Phase 3 clinical trial (VIITAL™) and an earlier Phase 1/2a study, with some patients being followed for up to eight years.
This milestone brings the RDEB community closer to an innovative new treatment option! Stay tuned for more updates.
