CHMP Endorses Krystal's Vyjuvek for Dystrophic Epidermolysis Bullosa

The European Medicines Agency’s (EMA) human medicines committee has recently recommended the approval of Krystal Biotech's Vyjuvek for treating dystrophic epidermolysis bullosa (DEB), a rare genetic disorder. The condition is severe, characterized by fragile skin, blistering, and scarring, often appearing at birth due to mutations in the collagen type VII alpha 1 chain (COL7A1) gene.

DEB significantly impacts patients' quality of life, as blisters and erosions can develop not only on the skin but also in areas such as the mouth, esophagus, rectum, and eyes. This can result in severe scarring and other critical health issues. Until now, care has primarily been palliative and supportive, with only one non-disease-modifying treatment available for this condition.

Vyjuvek represents a novel approach to treating DEB. It is a topical gene therapy applied weekly to wounds in small droplets, delivering new COL7A1 genes directly to the skin. This process aims to promote healing by actively addressing the genetic cause of the disease. The recommendation from the Committee for Medicinal Products for Human Use (CHMP) supports its use for patients of all ages, both in clinical settings and at home.

The decision is grounded in positive findings from a controlled trial involving 31 participants with an average age of 17. During the trial, each patient received Vyjuvek and a placebo gel on different wounds for comparison. Results indicated that Vyjuvek substantially improved wound healing, with 71% of wounds achieving complete healing at three months and 67% at six months, compared to only 20% and 22% with the placebo.

Suma Krishnan, president of research and development at Krystal Biotech, expressed enthusiasm about the CHMP’s recommendation. She highlighted the potential of Vyjuvek to become the first corrective therapy for DEB patients across Europe. Krishnan emphasized the importance of the therapy's wide-ranging label, which includes treatment from birth and allows for administration at home by patients or caregivers, thereby broadening access and minimizing barriers to starting and maintaining treatment.

The CHMP’s endorsement was facilitated through the EMA’s PRIority MEdicines scheme, which aims to provide additional scientific and regulatory support to treatments addressing significant unmet medical needs. The European Commission will now deliberate on the committee’s recommendation before making a final decision on the use of Vyjuvek for this indication.