FDA Greenlights ZEVASKYN™ as First-Ever Cell-Based Gene Therapy for RDEB

Abeona Therapeutics Inc. announced that ZEVASKYN™ has received U.S. Food and Drug Administration (FDA) approval. ZEVASKYN™ is the first and only autologous, gene-modified cell therapy approved for the treatment of wounds in both pediatric and adult patients with recessive dystrophic epidermolysis bullosa (RDEB). This drug is also known as prademagene zamikeracel or pz-cel.

RDEB is a severe and debilitating genetic skin disorder caused by mutations in both copies of the COL7A1 gene, preventing functional type VII collagen production. Without this protein and its anchoring fibrils, the skin becomes extremely fragile. This leads to chronic and non-healing wounds that are prone to infection. Previously, no FDA-approved treatment was available for RDEB. ZEVASKYN™ is now the only FDA-approved medication shown to heal RDEB wounds with a single application. This product delivers a functional COL7A1 gene into a patient’s skin cells using a retroviral vector, enabling the production of type VII collagen to support wound healing.

This approval was supported by results from the pivotal Phase 3 VIITAL™ clinical trial (NCT04227106). It is a multicenter, randomized, and intrapatient-controlled clinical study. This study met both co-primary efficacy outcomes and showed statistically significant wound healing of ≥50% from baseline and pain reduction from baseline as measured by the Wong-Baker FACES scale at 6 months after treatment. Among 43 chronic RDEB wounds treated with a single application of ZEVASKYN™, 81% achieved ≥50% wound healing (P<0.0001) compared to 16% of matched control wounds. Furthermore, <5% of patients reported the most frequent adverse events, such as itching and procedural pain.

In the Phase 1/2a clinical trial of ZEVASKYN (NCT01263379), a single-center, open-label trial was conducted with 38 chronic wounds across seven patients. A single surgical application of ZEVASKYN was associated with long-term improvement at treated sites. This improvement was observed during the median follow-up period of 6.9 years, ranging from 4 to 8 years.

“ZEVASKYN showed strong efficacy and tolerability with lasting wound healing and pain relief after a single application, offering a new treatment option for RDEB patients,” said Dr. Jean Tang, M.D., Ph.D., lead principal investigator of the VIITAL™ clinical study. “FDA approval of ZEVASKYN marks a breakthrough in treating RDEB and addressing the unmet need for chronic wound healing and pain reduction as shown by our pivotal Phase 3 clinical trial,” said Vish Seshadri, Ph.D., M.B.A., CEO, Abeona.

“After years of work, the FDA approval of ZEVASKYN is a great milestone for EB patients,” said Dr. M. Peter Marinkovich, M.D., co-principal investigator of the VIITAL™ trial. “We are confident in its ability to provide long-term results after a single treatment,” added Dr. Madhav Vasanthavada, Ph.D., M.B.A., Chief Commercial Officer, Abeona. Dr. Anna L. Bruckner, MD, Co-director, EB Clinic at Children’s Hospital, Colorado, and Professor of Dermatology, University of Colorado School of Medicine, agreed that ZEVASKYN offers a long-term treatment for RDEB and improves patients’ quality of life.

ZEVASKYN™ will be available in the third quarter of 2025 through Qualified Treatment Centers (QTCs) across the U.S., ensuring nationwide access to this important treatment at centers with EB and gene therapy expertise.

Reference: Abeona Therapeutics. U.S. FDA Approves ZEVASKYN™ (prademagene zamikeracel), the First and Only Cell-Based Gene Therapy for Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB). Published April 29, 2025. Accessed April 30, 2025. U.S. FDA Approves ZEVASKYN™ (prademagene zamikeracel), the First and Only Cell-Based Gene Therapy for Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) :: Abeona Therapeutics Inc. (ABEO)