FDA Approves First Cell-Based Gene Therapy For Rare Skin Disorder

Authored by Zachary Stieber via The Epoch Times (emphasis ours),

The Food and Drug Administration (FDA) has approved Zevaskyn, a gene therapy for a rare skin disorder, the company that makes the product said on April 29.

Regulators approved Zevaskyn for adults and children with recessive dystrophic epidermolysis bullosa, a disorder that leaves skin fragile and prone to blistering.

Severe cases of the disorder can result in loss of vision and other serious medical issues, according to the National Library of Medicine.

Recessive dystrophic epidermolysis bullosa has no cure.

Zevaskyn is the first cell-based gene therapy to receive approval for the condition. Abeona Therapeutics, which makes the therapy, said it only requires one application.

“Through a single surgical application, Zevaskyn can now offer people with [the condition] the opportunity for wound healing and pain reduction in even the most severe wounds,” Vish Seshadri, Abeona’s CEO, said in a statement.

Seshadri thanked participants in the company’s clinical studies, including a phase 3 trial that showed people who received the therapy experienced statistically significant improvement in healing, compared with a control group that received the standard of care.

Adverse events included itching.

“Zevaskyn was well-tolerated and efficacious in clinical studies, providing clinically meaningful improvements in wound healing, pain reduction, and other associated symptoms,” Dr. Jean Tang, a professor of dermatology who was the trial’s principal investigator, said in a statement.

Zevaskyn involves taking a patient’s skin cells and genetically modifying the cells to produce collagen. Up to 12 of the resulting cellular sheets are then surgically applied to a patient’s wounds.

The FDA did not return a request for comment.

Brett Kopelan, the executive director of Debra of America, which advocates for people with epidermolysis bullosa, expressed support for Zevaskyn, saying in a statement released by Abeona that the therapy “can significantly increase the quality of life of patients.”

Abeona said it expects Zevaskyn to be available starting in the third quarter of 2025. Patients seeking the therapy can receive it through Zevaskyn-qualified treatment centers.

About 3.3 per million people are affected by recessive and dominant dystrophic epidermolysis bullosa, according to the National Library of Medicine. The condition is caused by mutations in a gene called COL7A1. The mutations disrupt the body’s production of type VII collagen. That’s the collagen that Zevaskyn produces.

Two treatments are currently available. The Food and Drug Administration approved Vyjuvek, a gene therapy from Krystal Biotech, in 2023. The gel is applied to wounds regularly, typically once a week.

Filsuvez, also approved in 2023, and made by Chiesi Global Rare Diseases, can also be used. The gel, which contains birch bark, is also applied to wounds.