The US FDA has approved prademagene zamikeracel, an autologous cell sheet-based gene therapy, for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), a debilitating genetic skin disease. This is the first and only FDA-approved cell-based gene therapy to treat RDEB wounds with a single application. Clinical evidence In a clinical trial involving 11 patients with RDEB and a total of 86 wounds, 81% of wounds treated with prademagene zamikeracel achieved at least 50% healing, compared to only 16% in the standard of care group at six months.
Additionally, patients receiving prademagene zamikeracel experienced significantly greater pain reduction than controls, as measured by the Wong-Baker FACES scale (-3.07 vs. -0.90). Dosage and administration Available for topical application The recommended dose is based on the surface area of…
