FDA approval of prademagene zamikeracel (Zevaskyn) - advancing gene therapy for recessive dystrophic epidermolysis bullosa (RDEB) patients

Sep 29, 2026

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FDA approval on 29 April 2025 grants Zevaskyn (prademagene zamikeracel) authorization as a first-of-its-kind autologous, topical, cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB). RDEB is driven by COL7A1 mutations causing extreme skin fragility, recurrent blistering, chronic wounds, scarring, and cancer risk. Zevaskyn delivers functional COL7A1 via an HSV-1-derived vector into patient keratinocytes grown ex vivo as graftable tissue, improving wound closure and pain with minimal severe adverse events. The article also weighs durable benefits against a $3.1 million access barrier and calls for long-term surveillance.

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Highlights

FDA approval and mechanism

Clinical effects and safety

Access and cost considerations

Editorial FDA approval of prademagenezamikeracel(Zevaskyn): advancing gene therapy for recessivedystrophicepidermolysis bullosa (RDEB) patients Syeda Rabiah Shahid, MBBSa , Kamran Shahid, MBBSa , Maliha Khalid, MBBSb , Aminath Waafira, MBBSc ,* Dear Editor, On 29 April 2025, the U.S. Food and Drug Administration(FDA) approved Zevaskyn (prademagene zamikeracel) –a novel, first-of-its kind cell-based gene therapy – for RecessiveDystrophic Epidermolysis Bullosa (RDEB), transforming thetreatment of a rare and debilitating genetic skin disorder [1] .This autologous, topical, genetically modified cell-based therapyserves as an epitome of the advancements in targeted modernmedicine, offering a disease-modifying approach for a conditionformerly managed with only palliative measures. RDEB is an autosomal recessive rare disorder with an estimatedprevalence of 3.5–20.4 per million people worldwide[2] . The under -lying pathology of RDEB stems from mutations in the COL7A1gene which encodes collagen type VII – a critical component ofstructural fibrils that maintains dermal – epidermal cohesion andensures skin integrity. Therefore, its absence results in the hallmarkfeatures of RDEB, including extreme skin fragility, recurrent blis -tering, chronic non-healing wounds, progressive scarring, anda heightened risk of cutaneous squamous cell carcinoma[3] . The ensuing wounds are excruciatingly painful and can per -sist for years without healing. Despite the severity and predomi -nantly infantile patient population of the disease, the currentstandard of care is solely supportive, primarily via protectivebandaging and daily wound care. Thus, therapeutic regimensuntil now have failed to address the underlying defect or preventfuture injuries[4,5] Zevaskyn revolutionizes the therapeutic approach by deliver -ing functional COL7A1 into the patient’s own keratinocytes viaan HSV-1-derived vector. These genetically modified cells aregrown ex-vivo into graftable tissues, able to produce collagen aDepartment of Medicine, Shaikh Khalifa Bin Zayed Al-Nahyan Medical and DentalCollege, Lahore, Pakistan, bDepartment of Medicine, Jinnah Sindh MedicalUniversity, Karachi, Pakistan and cSchool of Medicine, The Maldives NationalUniversity, Malé, Maldives Sponsorships or competing interests that maybe relevant to the content aredisclosed atthe end of this article. *Corresponding author. Address: School of Medicine, The Maldives NationalUniversity, Malé 20026, Maldives. Tel.: +960 919-5504. E-mail: wafiagany@gmail.com(A. Waafira). Copyright © 2025 The Author(s). Published by Wolters Kluwer Health, Inc. This is anopen access article distributed under the Creative Commons Attribution License 4.0 (CCBY), which permits unrestricted use, distribution, and reproduction in anymedium, provided the original work is properly cited. Annals of Medicine & Surgery (2026) 88:47–48Received 29 May 2025; Accepted 20 November 2025Published online 2 December 2025http://dx.doi.org/10.1097/MS9.0000000000004501 # HIGHLIGHTS ● ZEVASKYN (Prademagenezamikeracel) isthe first FDA -approved autologous gene therapy for RDEB, targetingthe COL7A1 gene defect. ● A single topical application significantly improves woundhealing and reduces pain, with durable effects lastingmonths to years. ● Despite clinical success and a favorable safety profile, the$3.1 million cost presents a major barrier to accessibilityand equitable treatment. type VII. Subsequent application of these grafts over the woundsrestores epithelial integrity and promotes healing[6] . This recent FDA approval follows positive outcomes evident invarious clinical trials[7], includingthe crucial phase 3 VIITAL study:a multi-center, randomized, intra-patient-controlled trial. The trialexhibited a greater than 50 percent rate of wound closure, as wellas a marked reduction in pain scores from baseline within 6 months[8] . A single application of Zevaskyn – evidenced by theformer trial results – claims to

FAQs

What therapy did the FDA approve for RDEB on 29 April 2025?

The FDA approved Zevaskyn (prademagene zamikeracel) as an autologous, topical, cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB).

What genetic defect does Zevaskyn target, and how does it work?

Zevaskyn targets the COL7A1 gene defect. It delivers functional COL7A1 into the patient’s own keratinocytes using an HSV-1-derived vector, generating graftable tissues ex vivo to restore epithelial integrity and promote healing.

What benefits and safety profile were reported, and what major barrier remains?

The article reports improved wound healing and reduced pain with minimal severe adverse events, and local treatable infections. It also highlights that the $3.1 million per-treatment cost may limit accessibility and equitable treatment, especially for underinsured patients.

Sep 29, 2026

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