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 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.

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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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