FDA Approves Revolutionary Gene Therapy for Rare Skin Disorder

To this end, the U.S. Food and Drug Administration (FDA) last month approved Zevaskyn. This innovative, cell-based gene therapy is intended to provide a lasting cure for people living with recessive dystrophic epidermolysis bullosa (RDEB). This extreme genetic skin disease results in painful blistering and wounds. It happens due to the body’s extreme deficiency in the most important collagen type VII protein that’s vital for holding skin layers together. The approval represents an important step forward in providing treatment options for adults and children impacted by this debilitating condition.

Unlike other treatments, Zevaskyn uses a patient’s own skin cells. In the laboratory, these cells are genetically engineered to express the appropriate COL7A1 gene. This cellular fabric forming approach allows the easy production of a large, continuous sheet of cells. Surgeons could subsequently transplant it straight onto the patient’s wounds. This streamlined design makes Zivaskyn capable of treating nearly the entire affected area in a single procedure. This soft, glove-like material makes it a far-reaching solution for treating RDEB.

Clinical studies have demonstrated Zevaskyn to be effective. At six months, 81% of the typically difficult long-term, large wounds improved by at least 50%. In sharp contrast, just 16% of wounds treated with the conventional care reached comparable healing outcomes. These results further illustrate how Zevaskyn can have a transformative impact on patient outcomes.

“Grafting gene-corrected skin onto 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.” – Amy Paller, MD.

Zevaskyn is now the first and only treatment approved to treat the cause of RDEB. The device provides fresh optimism to people often dependent on conventional wound treatment and barrier dressing. A preliminary clinical study indicated that patients experienced long-term benefits from a single surgical application of Zevaskyn. Researchers followed up outcomes for almost seven years, many cases for up to eight years.

The therapy poses a serious risk of inducing cancer themselves, requiring all patients to be monitored for life after treatment. This unintended consequence of a liberating force exhibits the fundamental tension between delivering cutting-edge therapies and the enforcement of long-term patient welfare.