FDA Approves ZEVASKYN™, First Cell-Based Gene Therapy for RDEB

Abeona Therapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has granted approval for ZEVASKYN™, a pioneering gene-modified cellular therapy, as a treatment for both adult and pediatric patients suffering from recessive dystrophic epidermolysis bullosa (RDEB). This rare and severe genetic skin disorder currently has no cure, and ZEVASKYN emerges as the only FDA-approved treatment option that offers significant wound healing and pain reduction with just a single application.

ZEVASKYN’s approval was based on the notable outcomes of the Phase 3 VIITAL™ study. This trial was a multi-center, randomized, intrapatient-controlled investigation that successfully demonstrated statistically significant healing in more than 50 percent of large, chronic RDEB wounds, along with a reduction in pain assessed six months post-treatment. The study encompassed 43 large, chronic wounds treated with ZEVASKYN, 81 percent of which exhibited healing of 50 percent or more within six months, a stark contrast to just 16 percent in the control group treated with standard care.

From a safety perspective, ZEVASKYN was well-tolerated, with procedural pain and itch being the most common adverse events reported in less than five percent of patients. The underlying technology involves using the patient's own skin cells, specifically keratinocytes, genetically altered to produce functional Type VII collagen. This product is applied using sheets that can cover large wound areas, offering a substantial improvement for those affected by this debilitating condition.

The FDA approval of ZEVASKYN marks a significant milestone for individuals living with RDEB, providing a much-needed therapeutic option that promises to enhance the quality of life. The clinical evidence indicates that ZEVASKYN not only offers immediate benefits in wound healing but also suggests long-lasting effects. Earlier studies, such as the Phase 1/2a trial, showed that the benefits of a single application could last several years, providing renewed hope to those affected by this condition.

As a part of its deployment strategy, Abeona plans to make ZEVASKYN available through Qualified Treatment Centers by the third quarter of 2025. These centers are specialized facilities with expertise in cell and gene therapy, strategically located across the United States to maximize patient accessibility.

To support patients and families during their treatment journey, Abeona is offering a comprehensive support program called Abeona Assist™. This initiative provides personalized assistance, helping patients navigate insurance benefits, explore financial aid, and manage travel logistics. Such comprehensive support underscores the company's commitment to ensuring that eligible patients can access this innovative therapy seamlessly.

The significance of ZEVASKYN extends beyond just treating the symptoms of RDEB. It represents a groundbreaking advancement in the field of gene therapy, offering a new avenue of hope for individuals suffering from rare genetic disorders. The endeavor to address the challenges of RDEB with a single surgical application of ZEVASKYN highlights a critical step forward in therapeutic innovation.

In addition to the treatment benefits, Abeona has been granted a Rare Pediatric Disease Priority Review Voucher by the FDA, which underscores the therapy's potential to address a significant unmet medical need.

The approval of ZEVASKYN is not only a testament to the advances in gene therapy but also reflects the collaborative efforts of the scientific community, patients, families, and advocacy organizations committed to improving the lives of those affected by RDEB. With this new treatment option on the horizon, there is renewed optimism for those grappling with the challenges posed by this rare genetic condition.