The FDA approved Abeona Therapeutics' cell-based gene therapy, Zevaskyn (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa.

Prademagene zamikeracel (pz-cel) is approved for the treatment of adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). Patients with RDEB have markedly fragile skin, with recurrent blistering and chronic, nonhealing wounds. The disorder results from pathogenic variants in the COL7A1 gene, leading to deficient type VII collagen production and impaired dermal-epidermal adhesion.

Current standard of care consists primarily of supportive wound care and protective bandaging. Abeona Therapeutics anticipates commercial availability of Zevaskyn at designated treatment centers beginning in the third quarter of 2025.

Zevaskyn involves ex vivo transduction of autologous keratinocytes with a functional COL7A1 transgene, followed by grafting to chronic wounds. FDA approval was based on early- and late-phase clinical trial data demonstrating significant wound healing and pain reduction.

Existing gene therapy options include Vyjuvek (Krystal Biotech), approved in 2023 for the treatment of smaller wounds. Concurrent use of Vyjuvek and pz-cel may improve overall management of RDEB, according to Brett Kopelan, executive director of debra of America.