Topical gene therapy shows positive results in epidermolysis bullosa

Vyjuvek was effective in healing the majority of dystrophic epidermolysis bullosa wounds in a phase 3 trial, Krystal Biotech announced in a press release.

An investigational noninvasive topical gene therapy, Vyjuvek (beremagene geperpavec or B-VEC), delivers two copies of the COL7A1 gene to epidermolysis bullosa wounds.

The randomized, double-blind, intra-patient placebo-controlled phase 3 GEM-3 trial included 31 patients with primary matched wound pairs.

Complete wound healing, as assessed by investigators, was reported in 67% of those treated with Vyjuvek at 6-month timepoints, compared to 22% of those treated with placebo.

Complete wound healing at 3-month timepoints was reported in 71% of wounds treated with Vyjuvek, compared to 20% of placebo-treated wounds.

“Dystrophic epidermolysis bullosa is referred to as ‘the worst disease you’ve never heard of’ because of the incredibly devastating reality that patients with this genetic condition face, and we are thrilled to announce positive results from our pivotal GEM-3 trial of Vyjuvek which showed that this topical gene therapy led to durable wound healing in dystrophic EB wounds,” Suma Krishnan, founder and chief operating officer of Krystal, said in the release. “With these results in hand, we look forward to advancing discussions with regulatory authorities and will work quickly to bring this potential first-ever treatment to patients with dystrophic EB and their families who are in desperate need.”

B-VEK has been granted orphan drug designation by both the FDA and European Medicines Agency. The FDA has also given the drug fast-track designation and rare pediatric designation for the treatment of dystrophic epidermolysis bullosa.

Krystal plans to file a Biologics License Application with the FDA in the first half of 2022.