CHMP Backs Vyjuvek for Wound Care

Krystal Biotech, Inc. welcomed the European Medicines Agency's (EMA’s) announcement that its Committee for Medicinal Products for Human Use (CHMP) has issued a positive recommendation for the European Commission (EC) to approve Vyjuvek (beremagene geperpavec-svdt, or B-VEC) for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) who have mutations in the collagen type VII alpha 1 chain (COL7A1) gene, starting from birth. The CHMP’s positive opinion includes support for administration of Vyjuvek in either a health care setting (e.g., a clinic) or at home. If deemed appropriate by a healthcare professional, trained patients or caregivers may also apply Vyjuvek..

The final EC decision is anticipated in the second quarter of 2025. The decision will be applicable to all European Union member states, as well as Iceland, Norway and Liechtenstein.

“We are excited to be able to provide DEB patients with the first treatment that corrects the genetic defect and makes a true difference in their lives,” said Cristina Has, M.D., Professor and Head of the Genodermatoses Clinic in the Department of Dermatology at the University of Freiburg in Germany. “By addressing the very first stage in the complex pathophysiology of DEB, Vujuvek is a landmark. It is amazing how simple and non-invasive its use is, even in infants.”

“We are very pleased that our patients, from birth, will have a simple, topical treatment that promotes durable wound closure, something that until now has been beyond the reach of any therapy,” added Christine Bodemer, M.D., PhD, Professor and Head of the Department of Dermatology at the Necker Enfants Malades Hospital in Paris. “This is a remarkable advance for DEB patients and a new approach to gene therapy for genodermatoses, revolutionary and remarkably innovative.”

The positive opinion issued by the CHMP is based on a comprehensive clinical dataset including results from the Company’s Phase 1/II2 GEM-1 and Phase III GEM-3 studies, published in Nature Medicine and the New England Journal of Medicine, respectively (previously cited),  which collectively provided clear clinical evidence of successful COL7A1 gene delivery and durable wound closure following topical administration. The long-term safety and efficacy of B-VEC is further supported by results from the Company’s open label extension study completed in the United States as well as real-world experience with Vyjuvek since launching the United States in 2023.