May 19, 2026 —
The UK Medicines and Healthcare products Regulatory Agency has approved beremagene geperpavec, marketed as Vyjuvek by Krystal Biotech, as a topical gene therapy for wounds in patients with dystrophic epidermolysis bullosa, or DEB. The approval marks the first treatment of its kind for DEB patients of any age, with use permitted from birth onward.
DEB is a rare genetic skin disorder that causes fragile skin, recurrent blistering, and chronic wounds. The disease is caused by defects in the COL7A1 gene, which is responsible for producing type VII collagen, a protein needed to anchor the inner and outer layers of the skin together. Without sufficient functional type VII collagen, the skin becomes highly vulnerable to injury, blistering, and delayed wound healing.
Vyjuvek is applied once weekly directly to wounds as a gel. The therapy uses a modified, non-replicating herpes simplex virus type 1 vector to deliver two functional copies of the COL7A1 gene into skin cells. This allows treated cells to produce type VII collagen locally, helping support wound closure and reducing the risk of further skin damage.
Unlike permanent genome-editing approaches, Vyjuvek does not permanently alter the patient’s DNA. Because skin cells are naturally shed and replaced, the treatment is designed to provide a temporary but repeatable supply of the correct genetic instructions through ongoing topical administration.
Clinical evidence supporting approval included a Phase 3 double-blind, randomized, placebo-controlled trial in which patients received weekly treatment with either beremagene geperpavec or placebo for 26 weeks. At six months, 67% of wounds treated with Vyjuvek were completely healed, compared with 22% of placebo-treated wounds, demonstrating a meaningful improvement in wound healing.
The therapy was approved through the UK’s International Recognition Procedure, which allows the MHRA to consider assessments from trusted international regulatory partners. Following MHRA approval, a NICE decision on the clinical and cost effectiveness of Vyjuvek is expected in July.
The approval represents an important milestone for both DEB patients and the broader gene therapy field. As a topical, repeat-administered gene therapy, Vyjuvek offers a differentiated model for treating localized genetic disease and may help expand the therapeutic possibilities of gene delivery beyond systemic or one-time approaches.
