FDA approves Abeonas skin disease cell therapy
# FDA Approves ZEVASKYN:
First Cell-Based Gene Therapy for RDEB
Abeona Therapeutics has received FDA approval for ZEVASKYN (prademagene zamikeracel), marking a significant breakthrough in treating recessive dystrophic epidermolysis bullosa (RDEB), a rare and debilitating genetic skin disease14. This approval, announced on April 29, 2025, represents the first and only autologous cell-based gene therapy for treating wounds in both adult and pediatric RDEB patients1.
Key Facts
- ZEVASKYN (prademagene zamikeracel) is the first FDA-approved product to treat RDEB wounds with a single application13
- The therapy is designed for patients with RDEB, a serious genetic condition that causes severe wounds covering up to 30-80% of a person's body surface3
- Approval was based on the Phase 3 VIITAL study showing significant wound healing and pain reduction after a single treatment1
- In clinical trials, 81% of wounds treated with ZEVASKYN showed 50% or better healing after 6 months, compared to only 16% of wounds receiving standard care34
- The treatment will be available through Qualified Treatment Centers beginning in Q3 20251
How ZEVASKYN Works
ZEVASKYN uses an innovative approach to address the underlying genetic cause of RDEB. The therapy incorporates a patient's own skin cells that have been genetically modified using a retroviral vector to produce functional Type VII collagen34. These cells are then surgically applied as "sheets" over wounds3.
RDEB patients have mutations in both copies of the COL7A1 gene, which typically expresses Type VII collagen necessary for strengthening skin layers4. By restoring collagen VII expression at the wound site, ZEVASKYN effectively addresses a key aspect of the disease mechanism2.
Significance of Approval
This approval represents a major milestone for patients with RDEB, who previously had no FDA-approved treatments specifically targeting their condition1. The FDA also granted Abeona Therapeutics a Rare Pediatric Disease Priority Review Voucher as part of the approval1.
Abeona will support patients through their Abeona Assist™ program, which offers personalized support for eligible patients and families throughout their treatment journey1. This approval marks Abeona's entry into the commercial gene therapy arena after persistence through previous regulatory challenges3.
Sources:
1. https://investors.abeonatherapeutics.com/press-releases/detail/303/u-s-fda-approves-zevaskyn-prademagene-zamikeracel
2. https://www.biospace.com/press-releases/historic-achievement-fda-approval-of-abeona-therapeutics-zevaskyn-accelerates-momentum-and-expands-treatment-options-for-the-eb-community
3. https://www.fiercepharma.com/pharma/abeona-enters-commercial-gene-therapy-arena-fda-nod-zevaskyn-treat-rare-genetic-skin-disease
4. https://www.precisionmedicineonline.com/inherited-disease/fda-approves-abeonas-zevaskyn-ec-authorizes-krystals-vyjuvek-rare-skin-disease