US-based biotechnology company Krystal Biotech has received the European Commission (EC) marketing authorisation for Vyjuvek to treat a dystrophic epidermolysis bullosa (DEB)
Vyjuvek is a gene therapy indicated for the treatment of wounds in patients with DEB, with mutations in the collagen type VII alpha 1 chain (COL7A1) gene.
DEB is a genetic disorder that causes blisters and skin fragility due to a mutation in the collagen gene, specifically the COL7A1 gene.
Vyjuvek leverages a herpes-simplex virus type 1 (HSV-1) vector to deliver functional copies of the human COL7A1 gene.
The gene therapy aims to address the genetic root cause of DEB, promoting wound healing and sustained expression of type VII collagen protein.
It is the first corrective medicine approved for DEB in Europe, and offers flexible dosing options, allowing administration either at home or in healthcare settings.
The EC approval permits the marketing of Vyjuvek across all European Union (EU) member states, as well as Iceland, Liechtenstein, and Norway.
Krystal Biotech plans to launch Vyjuvek in Germany by mid-2025.
Krystal Biotech Europe general manager and senior vice president Laurent Goux said: “Today’s approval is an exciting milestone for Krystal and the patients we aim to serve.
“After years of preparation in close coordination with leading DEB treatment centres, we are ready and excited to bring this urgently needed therapy to patients.
“With already 1,000 DEB patients identified in France and Germany, as well as many more across the continent, our goal is clear, to ensure as many patients as possible are able to benefit from sustainable access to Vyjuvek.”
The EC approval follows positive recommendations from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).
It is supported by clinical data from Phase 1/2 GEM-1 and Phase 3 GEM-3 studies.
The studies showed successful COL7A1 gene delivery and durable wound closure, further confirmed by an open-label extension study and real-world data from the US.
Vyjuvek is already approved in the US and is under review by Japan’s Pharmaceuticals and Medical Devices Agency, with a decision anticipated in the second half of this year.
Krystal Biotech research and development president Suma Krishnan said: “We are delighted that Vyjuvek has been approved in Europe as a safe and effective therapy for the many patients across Europe suffering from DEB.
“This approval marks a critical milestone in our commitment to improving the lives of DEB patients around the world, and we look forward to providing patients in Europe with the first corrective medicine for this debilitating disease.”
