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The FDA has approved an updated label for beremagene geperpavec-svdt (B-VEC), a topical gene therapy for dystrophic epidermolysis bullosa (DEB), which expands the indication from ages 6 months and older to include newborns and makes treatment more convenient, according to a press release from manufacturer Krystal Biotech.
B-VEC, marketed as Vyjuvek, is currently approved in in the United States, Europe, and Japan as a topical treatment for wounds in patients with DEB. The manufacturer requested several updates to the current label to expand options for B-VEC, a genetically modified herpes-simplex virus designed to deliver normal copies of the COL7A1 gene — deficient in patients with DEB — to wounds to promote healing.
According to the approved updates, B-VEC can now be used at birth for children diagnosed with DEB, a genetic disorder that often presents at birth and affects the skin layers and causes chronic, painful wounds and blisters.
The updated label also allows patients with DEB and their caregivers to apply the treatment at home on their own and to remove wound dressings as part of the next dressing change, whenever it occurs, instead of the previously recommended set time of 24 hours — “further integrating Vyjuvek into existing wound care routines,” according to the press release. Previously, the treatment was administered by a healthcare professional in either a healthcare professional setting or at the patient’s home.
The updated approval was based in part on data from an open-label extension study in which patients treated with B-VEC on targeted wound areas for up to 112 weeks reported high levels of satisfaction and showed ongoing treatment response, with no new safety signals.
The treatment was originally approved by the FDA in 2023 for treatment of DEB in individuals aged 6 months and older, becoming the first FDA-approved gene therapy for DEB. Accompanying the revised label, updated application information allows patients to treat a wound under a blister cap after draining the blister, according to the manufacturer.
Full prescribing information includes details on dosage and safe handling.
