UK MHRA approves Krystal Biotech’s Vyjuvek for dystrophic epidermolysis bullosa wounds

Edited by Robert McTiernan··2 min read

Krystal Biotech (Nasdaq: KRYS) has received UK MHRA marketing authorization for Vyjuvek (beremagene geperpavec-svdt) for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) carrying mutations in the COL7A1 gene. The news marks the first genetic medicine approved in the UK for this condition.

The approval covers topical administration of Vyjuvek with flexible dosing options, including home administration by patients or caregivers, mirroring terms granted in the US, EU, and Japan. The therapy is designed to deliver functional copies of the COL7A1 gene via a replication-deficient herpes simplex virus type 1 (HSV-1) vector applied directly to wounds, enabling sustained type VII collagen expression and repeated dosing over time. Vyjuvek also qualifies for UK orphan designation, conferring up to 12 years of market exclusivity. Launch timing remains contingent on completion of reimbursement procedures.

The MHRA decision drew on data from the Phase I/II GEM-1 and Phase III GEM-3 studies, which together assessed topical COL7A1 gene delivery and wound closure in DEB patients. The Phase III GEM-3 trial used a within-patient controlled design comparing treated and untreated wounds, with complete wound closure as the primary endpoint. Results demonstrated durable wound closure following repeated topical application, supported further by an open-label extension and real-world experience with the product.

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The HSV-1 vector platform underpinning Vyjuvek allows the therapy to be redosed — a property that distinguishes it from one-time gene therapy approaches. Because DEB wounds are chronic and recurrent, the capacity for repeated topical application without immune preclusion is central to the therapy’s clinical rationale. The vector delivers COL7A1 transiently to keratinocytes at the wound site, with redosing intended to sustain collagen VII expression as wounds recur.

Vyjuvek enters a UK market where no gene-corrective therapy for DEB had previously been authorized. In the US, it competes with Zevaskyn (prademagene zamikeracel), approved by the US FDA in April 2025 for recessive DEB, which uses a one-time autologous cell sheet approach to restore COL7A1 function. Filsuvez (birch triterpenes topical gel), approved by the US FDA in December 2023 for partial-thickness wounds in DEB and junctional EB, addresses wound healing without correcting the underlying genetic defect. The UK approval is Krystal Biotech’s fourth regulatory clearance for Vyjuvek, following the US in May 2023, the EU in April 2025, and Japan in July 2025.

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