Abeona's Zevaskyn Gains FDA Approval for Rare Genetic Skin Disease, Marking Entry into Commercial Gene Therapy Arena

Abeona Therapeutics has achieved a significant milestone with the U.S. Food and Drug Administration (FDA) approving Zevaskyn, its autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB). This approval marks Abeona's entry into the commercial gene therapy market and provides a new treatment option for patients suffering from this debilitating genetic skin disease.

Zevaskyn: A Novel Approach to Treating RDEB

Zevaskyn, also known as prademagene zamikeracel, is designed to address the underlying genetic cause of RDEB. Patients with this condition have mutations in both copies of the COL7A1 gene, which is responsible for expressing Type VII collagen. The therapy involves genetically modifying a patient's own skin cells to produce functional Type VII collagen, which is then applied as a "sheet" over wounds.

The efficacy of Zevaskyn was demonstrated in the phase 3 VIITAL trial. After a single application, 81% of large and chronic wounds showed 50% or better healing at the 6-month mark. This is in stark contrast to the 16% healing rate observed in matched wounds treated with standard care.

FDA Approval and Commercial Launch

The FDA's approval comes approximately one year after initially rejecting Abeona's filing due to chemistry, manufacturing, and controls (CMC) issues. With these concerns now addressed, Abeona is preparing for a commercial launch in the third quarter of 2025.

Madhav Vasanthavada, Ph.D., Abeona's chief commercial officer, stated that the company plans to establish "outcome-based agreements" with commercial and government payers to ensure patient access to Zevaskyn. The therapy will be made available through qualified treatment centers across the United States.

To support the rollout, Abeona has established a manufacturing facility in Cleveland. The company also received a rare pediatric disease priority review voucher with the FDA approval, which it intends to monetize.

Competitive Landscape in Topical Gene Therapy

Abeona's entry into the commercial gene therapy market comes as competition in the field intensifies. Krystal Biotech, another player in the topical gene therapy space, launched Vyjuvek for dystrophic epidermolysis bullosa in May 2023. Vyjuvek has already generated over $341 million since its launch, indicating a strong market demand for innovative treatments in this area.

As Abeona prepares to enter this competitive landscape, the success of Zevaskyn will likely depend on its ability to demonstrate superior efficacy, safety, and ease of use compared to existing treatments. The company's focus on establishing partnerships with payers and treatment centers suggests a strategic approach to ensuring widespread access and adoption of the therapy.