Krystal Biotech Gains EU Approval for VYJUVEK® to Treat Dystrophic Epidermolysis Bullosa

Krystal Biotech, Inc. has received marketing authorization from the European Commission for its innovative therapy, VYJUVEK. This groundbreaking treatment targets dystrophic epidermolysis bullosa (DEB), a rare and severe genetic condition characterized by fragile skin prone to wounds. The approval signifies a significant step forward, allowing VYJUVEK to be used for patients with mutations in the collagen type VII alpha 1 chain (COL7A1) gene from birth. Importantly, this marks the first corrective therapy available in Europe for DEB patients.

VYJUVEK employs a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy to deliver functional copies of the human COL7A1 gene. This approach addresses the genetic cause of DEB, aiming to facilitate wound healing and sustain collagen protein expression with repeated doses. The therapy's flexibility in administration is notable, as it can be applied either at home or in a healthcare setting. Patients or caregivers may also administer the treatment if approved by a healthcare professional.

With this authorization from the European Commission, VYJUVEK can be marketed across all European Union member states, in addition to Iceland, Norway, and Liechtenstein. The timing of its availability will vary by country, contingent on the completion of reimbursement procedures. Krystal Biotech is preparing for its initial European release in Germany, anticipated in mid-2025.

Laurent Goux, Senior Vice President and General Manager of Europe at Krystal Biotech, expressed enthusiasm over the approval. He emphasized the company's readiness to introduce this much-needed therapy to patients. The company aims to provide widespread, sustainable access to VYJUVEK, with over 1,000 DEB patients already identified in France and Germany.

The European Commission’s decision follows a recommendation by the European Medicines Agency’s Committee for Medicinal Products for Human Use, based on comprehensive clinical data. This includes results from Krystal Biotech's Phase 1/2 GEM-1 and Phase 3 GEM-3 studies, which demonstrated successful gene delivery and durable wound closure. Further support came from the company's open-label extension study and real-world data from the United States.

Suma Krishnan, President of Research and Development at Krystal Biotech, highlighted the importance of this approval. She noted that it represents a crucial milestone in the company’s mission to improve the lives of DEB patients worldwide. Krystal Biotech is committed to providing European patients with this first corrective medicine for DEB.

VYJUVEK had already been approved by the FDA in the United States in May 2023. Furthermore, it is currently under review by Japan’s Pharmaceuticals and Medical Devices Agency, with a decision expected in the second half of 2025.

Krystal Biotech is a global biotechnology company focused on developing genetic medicines for diseases with significant unmet needs. VYJUVEK is its first commercial product, noted for being the first redosable gene therapy and the first genetic medicine approved for DEB by both the FDA and the EMA. The company continues to advance its pipeline of investigational therapies across various medical fields.

This latest approval by the European Commission not only expands the reach of VYJUVEK but also reinforces Krystal Biotech's commitment to delivering innovative treatments that address the root causes of genetic diseases.