Abeona Therapeutics Emerges in Dystrophic Epidermolysis Bullosa Market with ZEVASKYN Approval

Abeona Therapeutics Makes Significant Strides with ZEVASKYN Approval

In a pivotal move for the field of genetic dermatology, Abeona Therapeutics has achieved a major milestone with the FDA's approval of ZEVASKYN, an innovative autologous cell-based gene therapy. This breakthrough comes roughly a year after a previous approval attempt was turned down by the agency. The approval applies to both adult and pediatric patients suffering from recessive dystrophic epidermolysis bullosa (RDEB), a serious inherited skin disorder characterized by extreme skin fragility and frequent blister formation.

Understanding Dystrophic Epidermolysis Bullosa

Dystrophic epidermolysis bullosa is a subtype of a larger family of genetic disorders known as epidermolysis bullosa. It manifests through varying degrees of skin fragility and susceptibility to injuries, leading to complications that significantly impact the quality of life. The underlying cause is primarily related to deficiencies in proteins that provide structural integrity to the skin. As of 2023, there were an estimated 6,500 diagnosed cases of DEB across the seven major markets (7MM), with the U.S. accounting for roughly half of those.

While there remains no cure for this debilitating condition, comprehensive management strategies aim to alleviate symptoms through wound care, pain control, nutritional support, and infection prevention.

A New Player in the Market

With the introduction of ZEVASKYN, Abeona is strategically positioning itself in the commercial gene therapy market for dystrophic epidermolysis bullosa. Currently, the treatment landscape for DEB includes only four known therapies: the recently approved ZEVASKYN from Abeona, FILSUVEZ from Chiesi Farmaceutici, VYJUVEK from Krystal Biotech, and JACE from Japan Tissue Engineering. This limited availability indicates a substantial unmet therapeutic need.

Among the existing treatments, FILSUVEZ is notable for being a plant-based product and received EU marketing authorization in 2022. Both FDA and EMA have validated other therapies like VYJUVEK, which aims to deliver functional copies of the COL7A1 gene directly to DEB wounds. And Japan’s JACE has expanded its approval for both JEB and DEB.

Efficacy and Future Access to ZEVASKYN

The approval of ZEVASKYN (pz-cel) marks a significant advancement as it stands out as the first and only gene-modified cell therapy designed to treat wounds in patients with RDEB. This innovative therapy requires just a single application to be effective. The approval was based on promising outcomes from the Phase III VIITAL study, which demonstrated significant wound healing and pain reduction six months post-treatment.

Anticipated to hit the market in Q3 2025, ZEVASKYN will be available through certified treatment centers that specialize in gene and cell therapies, thus ensuring broader patient access.

Continued Innovation in DEB Treatment

Efforts to innovate continue, with a variety of promising therapies in the pipeline. Investigational therapies such as D-Fi (dabocemagene autoficel) and ABCB5+ mesenchymal stem cells are under development, aiming to revolutionize the treatment paradigm for DEB. D-Fi utilizes genetically engineered fibroblasts to produce type VII collagen, essential for skin structure. ABCB5+ MSCs are notable for their anti-inflammatory characteristics and potential to enhance tissue repair.

The ongoing research and development efforts are indicative of the growing need for effective DEB treatments. Forecasts predict the DEB market's growth from $550 million in 2023 to significant growth by 2034, driven by increasing prevalence, patient awareness, and a robust clinical pipeline.

Conclusion

The approval of ZEVASKYN signifies a critical step forward in the treatment of dystrophic epidermolysis bullosa. As new therapies emerge, they promise not only to enhance patient outcomes but also to catalyze economic growth and transform the DEB therapeutic landscape.