The FDA has granted approval for a groundbreaking cell-based gene therapy aimed at treating recessive dystrophic epidermolysis bullosa (RDEB), a rare and severe form of epidermolysis bullosa (EB), a genetic skin disorder that causes the skin to be extremely fragile. This condition leads to painful blisters and slow-healing wounds, which are prone to infections and complications over time. The newly approved treatment, Zevaskyn (prademagene zamikeracel or pz-cel), represents a significant advancement for those with RDEB, offering hope where previous treatment options have been limited to ongoing wound care and bandaging.
RDEB typically manifests in infancy and is caused by a mutation in the COL7A1 gene. This gene is responsible for producing collagen type VII, a crucial protein that binds the layers of skin together. In patients with RDEB, the faulty collagen results in skin that is excessively fragile, leading to the formation of blisters and open wounds that are difficult to treat. The condition is chronic, and affected individuals often experience extensive and painful skin damage throughout their lives.
The approval of Zevaskyn follows promising results from clinical trials, where it was found to provide substantial benefits in terms of wound healing and pain relief. In the studies, 81% of large, long-lasting wounds treated with a single application of Zevaskyn showed at least 50% healing after six months. This is a remarkable improvement compared to just 16% of wounds that were treated with standard care methods.
Zevaskyn is a novel therapy that involves the genetic modification of a patient’s own skin cells in a laboratory. These cells are engineered to incorporate a functional version of the COL7A1 gene, which allows them to produce the collagen necessary for proper wound healing. The modified cells are then cultured into a sheet that can be surgically applied directly to the patient’s wounds. This technique aims to cover a significant portion of the affected area in a single treatment, thereby promoting healing, reducing pain, and offering protection against infection.
The therapy has shown promising long-term results. An earlier study tracking patients for an average of seven years, and up to eight years in some cases, demonstrated that the benefits of a single application of Zevaskyn persisted over time. This long-term effectiveness is a significant breakthrough, as it suggests that the therapy could provide sustained relief from the chronic wounds and pain associated with RDEB.
Despite its promise, Zevaskyn is not without risks. The most common side effects are procedure-related pain and itching at the treatment site. Additionally, as with many gene therapies, there is a small risk of cancer, so patients who undergo this treatment will require lifelong monitoring. Side effects like severe itching, hives, difficulty breathing, and other allergic reactions are also possible, and patients are advised to seek immediate medical attention if they experience these symptoms.
The therapy is expected to become available in the third quarter of 2025 at specialized EB treatment centers across the United States. These centers, known as Qualified Treatment Centers, will be responsible for administering Zevaskyn to patients with RDEB. The approval of Zevaskyn marks a crucial step forward in the treatment of RDEB, offering a much-needed alternative for individuals who have suffered from the debilitating effects of this condition. The availability of this gene therapy represents a significant leap in the management of genetic skin disorders, providing hope for improved quality of life and long-term healing.
