FDA Approves Abeona's Gene Therapy for Rare Skin Disease, Marking Significant Advancement in Treatment

The U.S. Food and Drug Administration (FDA) has granted approval to Abeona Therapeutics' Zevaskyn (prademagene zamikeracel), a groundbreaking gene therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). This approval marks a significant milestone in addressing the unmet medical needs of patients suffering from this rare and debilitating genetic skin disorder.

Zevaskyn: A Novel Approach to RDEB Treatment

Zevaskyn, also known as pz-cel, is an autologous cell-based gene therapy that provides patients with genetically modified skin cells capable of producing functional Type VII collagen. The therapy is applied as a sheet over wounds, addressing the underlying cause of RDEB – mutations in the COL7A1 gene that lead to fragile, easily broken skin.

The FDA's decision was based on compelling data from the Phase III VIITAL study, which demonstrated significant wound healing and pain reduction in treated patients. According to the trial results, 81% of wounds in the treatment arm healed by 50% or more after six months, compared to only 16% in the placebo arm.

Commercial Launch and Market Impact

Abeona Therapeutics is preparing for a commercial launch of Zevaskyn in the third quarter of 2025. The company plans to make the treatment available through qualified epidermolysis bullosa treatment centers with prior cell and gene therapy experience, ensuring patient access across the United States.

In response to the approval, Abeona's stock initially surged by approximately 20% in early morning trading, though it later stabilized near its opening price of $5.35 per share. The company also received a rare pediatric disease priority review voucher, which it intends to monetize.

Addressing a Critical Unmet Need

RDEB is a severe condition characterized by chronic wounds that can persist for years, requiring frequent care and dressing changes. The disease has a devastating impact on patients' quality of life and longevity, with an 84% mortality rate by age 40.

Vish Seshadri, CEO of Abeona, emphasized the significance of this approval, stating, "We have heard from the RDEB community that there is a persistent unmet need to reliably address RDEB wounds, especially those that are chronic and prone to infection."

The approval of Zevaskyn represents a major advancement in the treatment of RDEB and highlights the growing potential of gene therapies in addressing rare genetic disorders. As the pharmaceutical industry continues to innovate in this space, patients with previously untreatable conditions may find new hope in these cutting-edge therapeutic approaches.