FDA Approves First Gene Therapy for RDEB

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Today, the US Food and Drug Administration (FDA) officially approved prademagene zamikeracel (pz-cel) (Zevaskyn; Abeona Therapeutics), marking a significant advancement for individuals living with recessive dystrophic epidermolysis bullosa (RDEB). Developed by Abeona Therapeutics, pz-cel is the first autologous, cell-based gene therapy specifically designed to address the genetic cause of RDEB, offering a new therapeutic option for a disease with historically limited treatment choices.¹

“Today’s approval of [pz-cel] represents a pivotal moment in the treatment of RDEB, answering the call of people living with the clinical, economic, and human impact of this devastating disease,” said Vish Seshadri, PhD, MBA, Chief Executive Officer of Abeona, in a news release. “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. Through a single surgical application, [pz-cel] can now offer people with RDEB the opportunity for wound healing and pain reduction in even the most severe wounds, as evidenced by the results from our pivotal phase 3 study.”

RDEB is a rare, inherited skin disorder characterized by mutations in the COL7A1 gene. This mutation disrupts the production of functional collagen VII, a critical protein needed to anchor the layers of the skin together. As a result, patients suffer from severe skin fragility, leading to chronic blistering, persistent wounds, debilitating pain, risk of life-threatening infections, and a heightened susceptibility to aggressive squamous cell carcinoma.

"In terms of diseases that require greater research and attention, conditions such as congenital melanocytic nevi, genetic skin diseases like epidermolysis bullosa and ichthyosis, and vascular anomalies associated with genetic syndromes remain areas of critical need," Mercedes E. Gonzalez, MD, co-chair of the Society for Pediatric Dermatology (SPD) Conference, said in a previous interview with Dermatology Times ahead of this year's meeting. "These rare disorders currently have no definitive cure, and increased research efforts could lead to improved treatments and better outcomes for affected children."

Pz-cel represents a novel therapeutic approach. It is created by harvesting a patient’s own skin cells, correcting the genetic defect ex vivo with a functional COL7A1 gene, and then expanding these corrected cells into sheets of keratinocytes. These gene-corrected cell sheets are surgically applied to chronic wound sites in a one-time procedure, where they express collagen VII locally, promoting more durable skin adhesion and wound healing.

"This is a very exciting therapy for patients with RDEB," Bernard Cohen, MD, professor of pediatrics and dermatology, Johns Hopkins University School of Medicine, told Dermatology Times sister publication Contemporary Pediatrics. "There have been few ways to effectively treat the skin erosions in these patients that may cover more than 30% of their skin surface at one time. Pz-cel allows for the patient's own skin cells to be genetically modified to produce functional Type VII collagen sheets that allow for rapid healing of ulcerated areas of skin. This allows for a dramatic improvement in the quality of life for these patients starting in early childhood," added Cohen.

The FDA’s approval decision was supported by a robust data package, including results from the pivotal phase 3 VIITAL study (NCT04227106) and a phase 1/2a study (NCT01263379). Clinical findings demonstrated significant and sustained wound healing after a single application of pz-cel. In some cases, benefits were observed for up to 8 years following treatment. Safety data from these trials indicated a favorable profile, with no new safety signals emerging during the long-term follow-up periods.²

“After many years of work, it is great to see this FDA approval of [pz-cel]. The EB patients deserve all that we can do for them,” said M. Peter Marinkovich, MD, associate professor of dermatology and co-principal investigator of the VIITAL study, in the release.

Seshadri added, “We are grateful to the patients, their families, and caregivers for their support of [pz-cel]. We express our gratitude to debra of America for their unwavering support throughout the development journey, in particular, for their interactions with the FDA in support of [pz-cel] and on behalf of the EB community that have helped make today’s regulatory approval possible. We are also thankful for the clinical study investigators, study site personnel, and the entire Abeona team for their collective commitment and determination through the development process, and contribution to this milestone achievement. We look forward to providing the RDEB community access to now-approved [pz-cel].”

The approval of pz-cell represents a historic step for RDEB patients and for the field of regenerative medicine. As the first autologous gene-corrected cell therapy approved for this indication, pz-cel may not only transform the clinical management of RDEB but also pave the way for future innovations in personalized, gene-targeted treatments.

In addition to its clinical promise, pz-cel has been recognized through multiple FDA designations, including Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations. These designations not only highlight the therapy’s potential but also underscore the urgent, unmet medical need for more effective RDEB treatments.

With the approval of pz-cel, Abeona Therapeutics may also be eligible to receive a Priority Review Voucher (PRV), a regulatory incentive designed to encourage the development of therapies for rare pediatric diseases.

Beyond pz-cel, Abeona Therapeutics is advancing a pipeline of gene therapies, including adeno-associated virus (AAV)-based programs targeting severe ophthalmic diseases. Their ongoing research and development efforts continue to focus on addressing unmet needs in rare, life-threatening conditions.

References

1. US FDA approves zevaskyn (prademagene zamikeracel), the first and only cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB). News release. Abeona Therapeutics. Published April 29, 2025. Accessed April 29, 2025. https://investors.abeonatherapeutics.com/press-releases/detail/303/u-s-fda-approves-zevaskyn-prademagene-zamikeracel
2. Abeona Therapeutics announces FDA acceptance of BLA resubmission of pz-cel for the treatment of recessive dystrophic epidermolysis bullosa. News release. Abeona Therapeutics. Published November 12, 2024. Accessed April 29, 2025. https://investors.abeonatherapeutics.com/press-releases/detail/294/abeona-therapeutics-announces-fda-acceptance-of-bla