FDA

Gene therapy approved for recessive dystrophic epidermolysis bullosa

May 2, 2025

Brand name: Zevaskyn

Generic name: prademagene zamikeracal

Manufacturer: Abeona Therapeutics

Approval date: April 29, 2025

FDA approved Zevaskyn (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB).

Zevaskyn is composed of autologous cells isolated from skin punch biopsies of patients with mutations in the collagen type VII alpha 1 chain (COL7A1) gene that have been transduced ex vivo with a replication incompetent retroviral vector (RVV) containing the full-length COL7A1 gene. The resulting gene-modified cell sheets express functional collagen VII (C7) protein.

Efficacy

Approval was based on the phase 3 VIITAL study (NCT04227106), a multi-center, randomized, intrapatient-controlled trial that met its two co-primary efficacy endpoints demonstrating statistically significant healing of ≥50% from baseline in large chronic RDEB wounds, and pain reduction from baseline as assessed by the Wong-Baker FACES scale, as evaluated at 6 months after treatment.

Across 43 large and chronic wounds treated with a single application of Zevaskyn, 81% of wounds showed ≥50% healing (P<0.0001) at six months, vs. 16% in 43 matched control wounds treated with standard of care.

Safety

The most common adverse events (<5% of patients) were procedural pain and itch.

Recommended dose

The recommended dosage of Zevaskyn in based on wound surface area. One sheet of Zevaskyn covers an area of 41.25 cm². Up to 12 Zevaskyn sheets can be joined together to cover large areas or applied to multiple distinct wounds.

Sources:

Zevaskyn (prademagene zamikeracel) [package insert]. Abeona Therapeutics. https://d1io3yog0oux5.cloudfront.net/_049178a312a1faf5ca9b3ff2103b92d0/abeonatherapeutics/files/ZEVASKYN_USPI_FINAL.pdf Revised October 2024. Accessed May 1, 2025.

US FDA approves Zevaskyn™ (prademagene zamikeracel), the first and only cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB). [News release]. 2025. https://investors.abeonatherapeutics.com/press-releases/detail/303/u-s-fda-approves-zevaskyn-prademagene-zamikeracel