FDA Approves Zevaskyn for Treatment of Rare, Genetic Skin Disorder

The U.S. Food and Drug Administration (FDA) has granted approval to Zevaskyn (prademagene zamikeracel), a groundbreaking gene therapy developed by Abeona Therapeutics, for patients suffering from recessive dystrophic epidermolysis bullosa (RDEB). This rare genetic skin disorder is characterized by extremely fragile skin that blisters and tears easily, causing significant discomfort and risk of infections.

Zevaskyn is notable for being the first and only autologous cell-based gene therapy designed to treat wounds in both adult and pediatric patients with RDEB. Its approval marks a significant advancement in the management of this debilitating condition.

The decision was supported by robust clinical data from two key studies. The first, a phase 1/2a open-label study involving seven patients with a total of 38 chronic wounds, showed that a single surgical application of Zevaskyn resulted in long-term improvements at treated sites over a median follow-up of seven years. The second, a multicenter, randomized phase III trial known as the VIITAL study, involved 43 large chronic wounds and demonstrated that 81% of wounds achieved at least a 50% healing rate six months after treatment. In comparison, only 16% of wounds treated with standard care showed similar healing.

Both studies confirmed that Zevaskyn was well tolerated, with no serious adverse events directly linked to the treatment. Common side effects included procedural pain and itching, affecting roughly 5% of patients.

Madhav Vasanthavada, Ph.D., chief commercial officer of Abeona Therapeutics, expressed confidence in Zevaskyn’s potential, citing the strong clinical evidence supporting its long-term efficacy after a single application. The company is also working towards outcome-based reimbursement agreements with payers to ensure broader access for patients.

This milestone offers new hope for those affected by RDEB, a condition that previously had limited treatment options, emphasizing the importance of ongoing research and innovation in genetic skin disorders.