FDA Approves Abeona’s Cell-Based Gene Therapy for RDEB

Rare Daily Staff

The U.S. Food and Drug Administration approved Abeona Therapeutics’ Zevaskyn, as the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with the rare skin condition recessive dystrophic epidermolysis bullosa.

The approval comes almost a year after Abeona initially anticipated a decision from the FDA because the agency requested additional information from the company in April 2024 to satisfy its requirements for chemistry, manufacturing, and control.

Recessive dystrophic epidermolysis bullosa (RDEB) is caused by a defect in the COL7A1 gene that results in the inability to produce type VII collagen. With mutations in both copies of the COL7A1 gene, people with RDEB have extremely fragile skin characterized by extensive blistering and severe wounds that often cover more than 30 percent of a patient’s body surface, and in some cases, up to 80 percent. RDEB wounds cause debilitating pain and systemic complications impacting the length and quality of life. These wounds are difficult to heal, can remain open for years, and many that do close tend to reopen.

Zevaskyn consists of a patient’s own skin cells (keratinocytes) that have been genetically modified, to produce functional type VII collagen. Zevaskyn sheets are surgically applied to the patient’s wounded areas. In a single application of Zevaskyn, up to 12 credit card-sized sheets can be joined together to cover large areas or applied to multiple distinct wounds, allowing for significant coverage of affected body areas. There is no cure for RDEB and Zevaskyn is the only FDA-approved product to treat RDEB wounds with a single application.

The FDA approval of Zevaskyn is based on the pivotal Phase 3 VIITAL study, a multicenter, randomized, intrapatient-controlled trial that met its two co-primary efficacy endpoints demonstrating statistically significant healing of 50 percent or more from baseline in large chronic RDEB wounds, and pain reduction from baseline as assessed by the Wong-Baker FACES scale, both evaluated at six months after treatment.

Across 43 large and chronic wounds treated with a single application of Zevaskyn, 81 percent of wounds showed 50 percent or more healing as evaluated at six months, compared to 16 percent in 43 matched control wounds treated with standard of care during the same period.

The most common adverse events were observed in fewer than 5 percent of patients and included procedural pain and itch.

Photo: Vish Seshadri, CEO of Abeona