Dystrophic Epidermolysis Bullosa (DEB) can be a very challenging condition to live with, & with the limited treatment options available on the market, which mainly works on treating the symptoms rather than solving the root cause of it, has made further more difficult to live with.

Dystrophic epidermolysis bullosa is ultra rare genetic blistering condition that happens primarily due to mutations in the COL7A1 gene which is responsible for producing a protein that helps in holding the layers of the skin together. It usually starts at birth & leads to extreme skin fragility, painful blisters & scarring. In it’s most severe cases DEB can affect eyes, mouth, internal organs causing life long complications & higher chance of developing skin cancer.

Vyjuvek offers a groundbreaking approach. It uses a modified herpes simplex virus to deliver a copy of the COL7A1 gene directly to wounds via a weekly topical application. This allows the skin to start healing at the genetic level, rather than simply managing symptoms.

The EMA has recommended grating marketing authorization in the European Union for Vyjuvek based on a clinical trial that was performed on 31 patients with dystrophic epidermolysis bullosa (DEB), including children as young as one year old, showed that weekly application of Vyjuvek significantly improved wound healing. After three and six months, 71% and 67% of Vyjuvek-treated wounds achieved full closure, compared to just 20% and 22% with placebo, demonstrating its strong potential as an effective treatment.

Moreover, Vyjuvek received support through EMA’s PRIME scheme for promising treatments that meet urgent medical needs.  After careful reviews both the community for advanced therapies (CAT) & the EMA’s Human Committee (CHMP) concluded the benefits of Vyjuvek outweighs the risk.

This breakthrough brings fresh hope and a brighter outlook for patients facing this tough condition

Learn more: First topical gene therapy treatment for dystrophic epidermolysis bullosa | European Medicines Agency (EMA)