Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa.
Shireen V GuideMercedes E GonzalezI Sinem BağcıBrittani AgostiniHubert ChenGloria FeeneyMolly SteimerBinoy KapadiaKunju SridharLori Quesada SanchezFranshesca GonzalezMatthew Van LigtenTrevor J ParrySurya ChitraLisa A KammermanSuma KrishnanM Peter MarinkovichPublished in: The New England journal of medicine (2022)
Complete wound healing at 3 and 6 months in patients with dystrophic epidermolysis bullosa was more likely with topical administration of B-VEC than with placebo. Pruritus and mild systemic side effects were observed in patients treated with B-VEC. Longer and larger trials are warranted to determine the durability and side effects of B-VEC for this disease. (Funded by Krystal Biotech; GEM-3 ClinicalTrials.gov number, NCT04491604.).