Vyjuvek shows dystrophic epidermolysis bullosa wound healing
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Patients with dystrophic epidermolysis bullosa had positive phase 3 results with Vyjuvek treatment, Krystal Biotech announced in a press release.
Vyjuvek (investigational beremagene geperpavec) is a noninvasive, topical, redosable gene therapy.
The randomized, double-blind, intra-patient, placebo-controlled phase 3 GEM-3 trial enrolled 31 patients with dystrophic epidermolysis bullosa (dystrophic EB) whose wounds were randomly assigned to receive either weekly Vyjuvek or placebo.
Complete wound healing as assessed by investigators occurred in 67% of the wounds treated with Vyjuvek, compared with 22% of those treated with placebo, at 6 months.
In addition, 71% of the wounds treated with the gene therapy achieved complete wound healing in 3 months, compared with 20% of those in the placebo treatment group.
“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.”
Krystal Biotech plans to apply for a Biologics License Application with the FDA and a Marketing Authorization Application with the European Medicines Agency based on these results.
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