The condition, recessive dystrophic epidermolysis bullosa, causes chronic wounds and has an 84% mortality rate by age 40.
Abeona got the FDA go-ahead Tuesday for its cell-based gene therapy prademagene zamikeracel for the rare skin disorder recessive dystrophic epidermolysis bullosa. The treatment, also known as pz-cel, will be marketed as Zevasykn.
The Cleveland-based company’s stock jumped about 20% in early morning trading but has since returned to near its opening price of $5.35/share.
Recessive dystrophic epidermolysis bullosa (RDEB) is a rare but debilitating condition characterized by fragile, easily broken skin, resulting from a mutation in the COL7A1 gene that encodes a component of collagen. Patients with the condition require frequent care and dress changes for wounds that can persist for years. According to an email from the company sent to BioSpace, RDEB kills some 84% of patients by age 40.
Zevasykn’s approval is based on data from a Phase III VIITAL study, where 81% of wounds in the treatment arm had healed by 50% or more, compared to 16% in the placebo arm, as well as pain reduction when re-assessed six months post-treatment. Procedural pain and itching were the most common adverse effects.
“We have heard from the RDEB community that there is a persistent unmet need to reliably address RDEB wounds, especially those that are chronic and prone to infection,” Abeona CEO Vish Seshadri said in a statement.
Zevasykn is an autologous sheet-based gene therapy that provides patients with cells carrying a correct version of the COL7A1 gene, which are surgically applied in credit card–sized sheets to a patient’s wounds. The company expects the treatment to be available by the third quarter of 2025 through epidermolysis bullosa treatment centers with prior cell and gene therapy experience.
On the heels of the approval, Abeona received a rare pediatric disease priority review voucher, which the company plans to monetize.
