Exciting news: US-based Krystal Biotech announces positive results from GEM-3 Regulatory Study for VYJUVEKTM
VYJUVEKTM is a non-invasive and topical gene therapy for dystrophic EB. It is currently the only gene-targeting approach for treating dystrophic EB that has successfully completed a Phase 3 trial.
The primary endpoint of the study examined wound healing of topical VYJUVEKTM compared to placebo at six months. The study reached statistical significance. Thirty-one EB patients were included in the study. At three months, 71% of wounds treated with the agent achieved complete wound closure, compared with 20% of wounds treated with placebo. At six months, 67% of wounds treated with the agent achieved complete wound closure compared with 22% of wounds treated with placebo.
The treatment is transient and requires a repeated application. As a topical product, it is a more patient-friendly treatment and can treat large areas without trauma. The approach sounds very promising and will soon seek approval in Europe as well.