Abeona Sells Priority Review Voucher for $155 Million

Rare Daily Staff

Abeona Therapeutics said it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher for $155 million.

The U.S. Food and Drug Administration awarded Abeona the Priority Review Voucher (PRV) following the approval of Zevaskyn, the company’s cell-based gene therapy for the treatment of wounds in adults and pediatric patients with the rare skin condition recessive dystrophic epidermolysis bullosa (RDEB).

Zevaskyn is the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with RDEB.

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 both 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 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 combined 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.

“With proceeds from this PRV sale, we have sufficient cash for more than two years of operating expenses without the need for capital infusion and not accounting for Zevaskyn sales,” said Joe Vazzano, chief financial officer of Abeona. “Furthermore, with Zevaskyn becoming available to treat patients beginning third quarter of 2025, we anticipate becoming profitable in early 2026.”

The sale comes as the future of the Rare Pediatric Priority Review Voucher program remains uncertain. The industry and rare disease community have lobbied to renew the program because it provides a needed incentive for developing these therapies. It has also proved to be an important source of capital at a time when companies have faced challenges raising money.

The program was set to end on September 30, but it was extended until December 20. There is proposed legislation that would renew it through September 30, 2030.

The Priority Review Voucher can be used to shorten the FDA review time of a therapy seeking marketing approval to six months from the usual 10 months. Since the program’s expiration, the price for PRVs has hovered around $150 million.