The FDA has approved a new cell-based gene therapy for people with a rare genetic skin disorder known as recessive dystrophic epidermolysis bullosa (RDEB), after clinical studies showed significant and longer-lasting benefits from a single treatment.
The treatment does, however, carry a small risk of cancer, according to the listed side effects.
Epidermolysis bullosa (EB) affects about 200 children every year in the United States, with RDEB being the more severe form. The treatment, known as prademagene zamikeracel or pz-cel but sold as Zevaskyn, is the first and only approved treatment of its kind for RDEB, offering hope to both adults and children who usually rely on regular wound care and protective bandaging.
WebMD reports that RDEB is a lifelong condition that usually appears in infancy. It is caused by a faulty COL7A1 gene, which normally helps produce a type of collagen that holds the layers of skin together.
When this collagen is defective, the skin becomes extremely fragile, leading to painful blisters and wounds that are slow to heal, prone to infection, and may result in serious health complications over time.
The FDA’s decision to approve the therapy was based on a clinical trial that showed Zevaskyn was well-tolerated and helped with wound healing and pain relief in patients with RDEB, according to Abeona Therapeutics, Zevaskyn’s maker.
After six months, 81% of large long-lasting wounds treated with a single application of Zevaskyn showed at least 50% healing, compared with only 16% of wounds treated with standard care.
The company also reported that an earlier study showed long-term benefits after a single surgical application of Zevaskyn, with results tracked for nearly seven years on average – and up to eight years in some cases.
Zevaskyn uses a patient’s skin cells that are genetically modified in a lab to add the correct COL7A1 gene. This allows the cells to produce the functional version of collagen protein needed for proper wound healing.
Zevaskyn is designed as a sheet of cells that can be surgically transplanted onto the wounds, covering most of the affected area in a single procedure. This helps the wounds to heal, reduces pain, and protects them from infections.
Amy Paller, MD, a paediatric dermatologist and clinical researcher, said the new therapy would be an effective addition to other available therapies.
“Grafting gene-corrected skin on to chronically open wounds of patients with recessive dystrophic epidermolysis bullosa promises the potential to provide long-term healing of wounds, reduction in pain and reduced risk of infection,” she said.
The most common side effects include procedure-related pain and itching.
Zevaskyn also carries a small risk of causing cancer, so patients may need lifelong monitoring, according to the safety information from Abeona Therapeutics.
The company expects Zevaskyn to be available in the third quarter of 2025 through specialised EB treatment centres, known as Qualified Treatment Centres, across the US.
WebMD article – FDA Approves New Gene Therapy for Rare Genetic Skin Disorder (Open access)
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