Krystal Biotech Reports Positive Topline Results from Phase 3 Trial of Beremagene Geperpavec for Dystrophic Epidermolysis Bullosa

Krystal Biotech reported positive topline results from the pivotal phase 3 GEM-3 trial of beremagene geperpavec (B-VEC), now known as Vyjuvek, for the treatment of dystrophic Epidermolysis Bullosa (dystrophic EB). The trial met the primary endpoint of complete wound healing at six-month timepoints and the secondary endpoint of complete wound healing at three-month timepoints. The company is on-track to file a Biologics License Application (BLA) with the U.S. FDA in the first half of 2022.

GEM-3 is a randomized, double-blind, intra-patient placebo-controlled study and enrolled 31 patients with dystrophic EB. The patients ranged in ages from one (1) year to forty-four (44) years old.

In each patient, a primary wound pair was identified by the investigator; one wound was randomized to receive a weekly topical application of Vyjuvek and the other to receive placebo. Wounds were dosed once-weekly with either Vyjuvek or placebo until closure. Weekly application was resumed if wounds re-opened at any point in the study. The primary outcome measure was complete wound healing determined by the Investigator in Vyjuvek treated wounds versus placebo treated at the six-mon...

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