This week marked the first time a patient with recessive dystrophic epidermolysis bullosa (RDEB) received prademagene zamikeracel (pz-cel, Zevaskyn), a first-of-its-kind autologous gene therapy to treat wounds in children and adults with the disease, according to a press release from the manufacturer Abeona Therapeutics.

The treatment, approved by the FDA in April of this year, was administered at the Lucile Packard Children’s Hospital Stanford in Palo Alto, California, on December 8.

The therapy uses skin cells from a patient’s skin plus animal material to create sheets that are surgically applied to wounds. This“incorporates the functional type VII collagen-producing COL7A1 gene into a patient’s own skin cells, ex vivo, using a replication-incompetent retroviral vector to produce functional type VII collagen in treated wounds,” according to the company. RDEB, caused by a defect in the COL7A1 gene, is a rare, debilitating genetic skin disease with no cure, and patients have extremely fragile skin often with large open wounds that can stay open for years with a high risk for systemic infection.

Pz-cel is the only FDA-approved product to treat RDEB wounds with a single application. Two other EB-specific therapies have been approved since 2023: a topical gene therapy, Vyjuvek, administered once a week and a topical birch bark extract-based agent, Filsuvez, applied to wounds until the wounds are healed.

“We continually pursue new therapies to support patients enduring the lifelong pain and extensive wound care that RDEB demands and work to provide care and hope to patients and families,” Joyce Teng, MD, PhD, professor of dermatology at Stanford Medicine and chief of pediatric dermatology at Stanford Medicine Children’s Health, said in the Abeona press release. Teng was the lead investigator on the trial that led to the FDA approval of pz-cel.

In October, Abeona announced that Children’s Hospital of Colorado had become the third qualified treatment center for pz-cel for RDEB, following the Stanford site and the Ann & Robert H. Lurie Children’s Hospital of Chicago.

The company has said that the list price for the one-time treatment would be $3.1 million for every 12 sheets produced, each sheet about the size of a credit card.

Patient labeling cautions that patients may experience pain or itch at the wound site as the area heals. Instructions for patients include not disturbing the dressing for 5-10 days unless a provider instructs otherwise. Patients should contact providers if they have increased drainage, worsening pain or swelling, or any other symptoms at or near the wound site. Dressings must be kept dry until the gauze backing falls off the treated area or areas about 2-3 weeks after surgery.

Safety information also includes a potential risk that the treatment may contribute to the development of cancer. “Patients should be monitored for the rest of their lives to check for any signs of cancer,” the information states.

Pz-cel’s approval was based on the multicenter, randomized, intrapatient-controlled phase 3 VIITAL study of patients aged 6 years or older with RDEB, which showed pz-cel resulted in significant wound healing and reduced pain after one treatment.