NEW YORK – The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) on Friday recommended the approval of Krystal Biotech's gene therapy Vyjuvek (beremagene geperpavec) to treat wounds among patients with dystrophic epidermolysis bullosa (DEB), a rare genetic disease.
The committee based its decision in part on data from the Phase I/II GEM-1 and Phase III GEM-3 clinical trials of Vyjuvek, according to Pittsburgh-based Krystal. In the Phase III trial, 31 DEB patients, including 19 pediatric patients, had one wound treated with Vyjuvek and a matched wound with placebo.
Vyjuvek, a gene therapy that is delivered topically once a week as a gel, uses a genetically modified herpes-simplex virus type 1 vector to deliver two functional copies of COL7A1 to patients' wounds. Mutations in COL7A1 lead to DEB, a condition in which patients have fragile skin that blisters and tears easily and leads to debilitating scarring. At six months, treatment with Vyjuvek led to complete healing in 67 percent of wounds, compared to 22 percent of those administered placebo.
"The CHMP's recommendation for approval of Vyjuvek is an exciting step towards our goal of delivering the first ever corrective therapy to DEB patients across Europe," Suma Krishnan, president of R&D at Krystal Biotech, said in a statement, further noting that CHMP's recommendation is for DEB patients of all ages and supports administration of Vyjuvek both in a healthcare setting or at home.
CHMP's recommendation will now be reviewed by the European Commission, and a decision is expected in the second quarter of the year. Krystal said it is on track to launch Vyjuvek in Germany in the middle of the year and in France later in 2025.
Vyjuvek has already been approved in the US.