The phase III GEM-3 trial of Vyjuvek meets primary endpoint in dystrophic epidermolysis bullosa.
The pivotal study included 31 patients with dystrophic epidermolysis bullosa who had one wound treated weekly with a topical application of Vyjuvek while another was treated with placebo. The study's primary outcome measure was complete wound healing at six-month, while secondary goals included investigator-assessed complete wound healing at the three-month mark and mean change in pain severity.
According to top-line results, data showed that 67% of wounds treated with Vyjuvek achieved the primary endpoint, compared to 22% of wounds treated with placebo. At the same time, 71% of wounds treated with the gene therapy achieved the secondary endpoint of wound healing at the three-month limit, versus 20% of wounds treated with placebo. Results show that Vyjuvek was well tolerated, with only one mild drug-related adverse event reported. In addition, the immunogenicity profile of the gene therapy, that delivers two copies of the COL7A1 gene, was consistent with previous research and no meaningful changes in anti-HSV-1 or anti-COL7 antibodies were seen.
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