Krystal Biotech’s VYJUVEK Receives Positive Recommendation from European Medicines Agency
PITTSBURGH, Feb. 28, 2025 – Krystal Biotech, Inc. (KRYS), a leading commercial-stage biotechnology company, announced with great enthusiasm the European Medicines Agency’s (EMA) recent decision. The Committee for Medicinal Products for Human Use (CHMP) of the EMA 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. This recommendation covers the administration of VYJUVEK in either a healthcare setting or a homecare setting.
Understanding Dystrophic Epidermolysis Bullosa (DEB)
Dystrophic epidermolysis bullosa (DEB) is a rare, severe, and often life-threatening genetic disorder that affects the integrity of the skin and mucous membranes. Patients with DEB experience fragile skin that blisters and tears easily, leading to chronic wounds that are prone to infection. The disease is caused by mutations in the COL7A1 gene, which results in the deficiency or absence of type VII collagen, a crucial component of the anchoring fibrils that attach the epidermis to the underlying dermis. This deficiency results in the blistering and fragility of the skin.
The Impact of VYJUVEK on Patients with DEB
VYJUVEK, an autologous cell and tissue-based therapy, is a significant breakthrough for patients with DEB. The treatment utilizes the patient’s own cells, which are then genetically modified to produce functional type VII collagen. Once the cells have been modified, they are applied to the patient’s wounds, promoting the growth of new, healthy skin. With the positive recommendation from the EMA, VYJUVEK will soon be available to eligible patients in Europe, providing them with a much-needed treatment option.
Homecare Administration of VYJUVEK
The CHMP’s positive opinion includes support for the administration of VYJUVEK in a homecare setting. This is a significant development as it will enable patients to receive the treatment in the comfort of their own homes, reducing the need for frequent hospital visits. The homecare administration will not only improve patient convenience but also potentially reduce healthcare costs by decreasing the need for hospital stays and travel expenses.
The Global Impact of VYJUVEK
The positive recommendation from the EMA is a major milestone for Krystal Biotech and the DEB community. With this approval, VYJUVEK will be available to patients in the European Union, and the company is actively working on gaining approval in other regions, including the United States and Japan. This global expansion of VYJUVEK has the potential to significantly impact the lives of thousands of DEB patients worldwide.
Conclusion
The European Medicines Agency’s positive recommendation for VYJUVEK represents a significant step forward in the treatment of dystrophic epidermolysis bullosa (DEB), a rare and debilitating genetic disorder. The autologous cell and tissue-based therapy, which utilizes the patient’s own cells to produce functional type VII collagen, is now one step closer to being available to eligible patients in Europe. With the potential for homecare administration and global expansion, VYJUVEK has the power to transform the lives of thousands of DEB patients worldwide, providing them with a much-needed treatment option and improving their overall quality of life.
- Krystal Biotech’s VYJUVEK receives positive recommendation from European Medicines Agency
- Treatment for wounds in DEB patients with COL7A1 gene mutations
- Homecare administration of VYJUVEK approved in Europe
- Potential global impact on thousands of DEB patients
