Abeona Therapeutics Wins FDA Approval For First-of-Its-Kind Gene Therapy

The U.S. Food and Drug Administration (FDA) on Tuesday approved Abeona Therapeutics Inc.’s (NASDAQ:ABEO) Zevaskyn (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the first and only autologous cell-based gene therapy for wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB).

RDEB is a severe, genetically inherited skin disorder characterized by blistering and skin fragility, often presenting at birth.

Zevaskyn is the only FDA-approved product to treat RDEB wounds with a single application.

Zevaskyn consists of a patient’s skin cells (keratinocytes) genetically modified to produce functional Type VII collagen. Zevaskyn sheets are surgically applied to the patient’s wounded areas. In a single application, up to 12 credit card-sized sheets can be combined to cover large areas or applied to multiple distinct wounds, allowing for significant coverage of affected body areas.

Also Read: Incyte’s New Treatment for Chronic Skin Disease Shows Promising Results In Latest Clinical Trials, Stock Falls

Zevaskyn is expected to be available beginning in the third quarter of 2025 through Zevaskyn Qualified Treatment Centers.

The FDA approval is based on the pivotal Phase 3 VIITAL study, which met its two co-primary efficacy endpoints, demonstrating statistically significant healing of 50% or more from baseline in large chronic RDEB wounds and pain reduction from baseline as assessed by the Wong-Baker FACES scale, as evaluated at six months after treatment.

Across 43 large and chronic wounds treated with a single application of Zevaskyn, 81% of wounds showed 50% or more healing (P<0.0001) as evaluated at six months, compared to 16% in 43 matched control wounds treated with standard-of-care.

The most common adverse events were observed in fewer than 5% of patients, including procedural pain and itch.

The Phase 1/2a study of Zevaskyn, which involved 38 chronic wounds across seven patients, showed that a single surgical application of Zevaskyn was associated with long-term improvement at treated sites over a median follow-up of 6.9 years, ranging from 4 to 8 years.

Last April, the FDA issued a Complete Response Letter (CRL) in response to the company’s Biologics License Application (BLA) for pz-cel for RDEB. 

The FDA noted that certain additional information was needed to satisfy Chemistry Manufacturing and Controls (CMC) requirements. 

Price Action: ABEO stock is up 2.34% at $5.46 at the last check Tuesday.

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