FDA Approves First Cell-Based Gene Therapy for Rare Genetic Skin Disorder
May 05, 2025
The Food and Drug Administration recently
approved prademagene zamikeracel (Zevaskyn, Abeona Therapeutics), the first cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB), a rare and debilitating genetic skin disorder.
RDEB, also known as “butterfly skin disease,” is caused by biallelic mutations in the COL7A1 gene, resulting in absent or defective Type VII collagen. Patients develop extensive blistering and chronic wounds, often affecting more than 30% of the body surface.
Prademagene zamikeracel is a one-time therapy that uses genetically modified autologous skin cell sheets to promote wound healing and reduce pain in patients with RDEB.
The approval was based on the
phase 3 VIITAL trial, in which 81% of large chronic wounds treated with prademagene zamikeracel achieved at least 50% healing at six months, compared with 16% of wounds receiving standard care. The therapy was well tolerated, with no treatment-related serious adverse events.
Abeona expects prademagene zamikeracel to be available through qualified treatment centers beginning in the third quarter of 2025.
