First topical gene therapy treatment for dystrophic epidermolysis bullosa

EMA has recommended granting a marketing authorisation in the European Union (EU) for Vyjuvek (beremagene geperpavec) to treat wounds in patients of all ages with dystrophic epidermolysis bullosa (DEB).

Dystrophic epidermolysis bullosa is a serious, ultra-rare genetic blistering disease caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene. This gene is involved in the production of type VII collagen (COL7), a protein crucial in supporting and structuring the layers of the skin. DEB is characterised by skin fragility, blister formation, small white bumps (milia) and scarring, and is usually present at birth. Blisters and erosions may also develop in the mouth, oesophagus, rectum, genitourinary system and eyes, or other mucous tissue. Healing of blisters and erosions can result in debilitating scarring and other serious conditions. Recessive DEB, the most severe form of DEB, can lead to the loss of fingernails and toenails, joint deformities, vision loss and unusually aggressive squamous cell carcinoma (a type of skin cancer).

More information on the EMA Website.

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