FDA expands B-VEC label to include patients from birth with dystrophic epidermolysis bullosa

On September 15, 2025, the FDA expanded the beremagene geperpavec-svdt (B-VEC; Vyjuvek; Krystal Biotech) indication to include patients from birth with dystrophic epidermolysis bullosa (DEB), expanding on the previous indication of 6 months or older.¹

Originally approved by the FDA in May 2023, B-VEC can now provide full flexibility with respect to application and management of wound dressings, according to a press release from Krystal Biotech. In addition to allowing patients with DEB and caregivers to apply B-VEC at home and on their own, the label update allows wound dressings to be removed as part of the next dressing change rather than waiting 24 hours.

"This just represents an immense move forward in terms of actual rational design of a treatment where we can actually make a big difference in individuals' lives," said Raj Chovatiya, MD, PhD, in a previous interview with Contemporary Pediatrics at the time of the original B-VEC approval.²

“We believe these changes further reinforce [B-VEC's] leadership position as the most flexible, convenient, and disease-modifying medicine to treat DEB patients in the United States,” said Krish S. Krishnan, chairman and CEO of Krystal Biotech, of the updated label. "Overall, we believe that these advancements solidify [B-VEC] as the standard of care for all DEB wounds, regardless of wound size and severity, and will further improve compliance.”¹

Long-term extension study of B-VEC

Approval of the updated label was based on real-world data collected since the topical's launch in the United States in addition to results from the open-label extension study (NCT04917874) conducted in the US earlier in 2025. Results reinforced efficacy and safety of B-VEC across ages, including in cases of patient or caregiver application.^1,3

A total of 47 patients, including 24 rollover participants from the phase 3 trial and 23 treatment-naive individuals, received weekly applications of B-VEC to target wound areas for up to 112 weeks (median, 81 weeks). Safety assessments focused on adverse events, whereas exploratory outcomes included treatment satisfaction, quality of life, and wound closure rates among rollover participants.³

Overall, 74.5% of patients experienced at least 1 adverse event, the majority of which were mild or moderate in severity. Seventeen serious adverse events were reported in 14 patients, and 14 severe adverse events were noted in 10 patients; however, none were deemed treatment related. No adverse events led to study discontinuation. Patient-reported outcomes suggested consistently high levels of treatment satisfaction, though quality-of-life measures yielded inconclusive results.

WATCH: How FDA-approved B-VEC will help children with dystrophic epidermolysis bullosa, with Bernard A. Cohen, MD

Wounds that had been treated with B-VEC during the phase 3 study maintained high rates of closure throughout the extension, ranging from 61.1% to 89.5% between baseline and month 12. These findings reinforce the long-term safety profile of B-VEC and its ability to sustain clinical benefit over extended treatment periods. Although the open-label design and variable follow-up remain limitations, the data support the continuous use of B-VEC for DEB management.

"The updates to the [B-VEC] label are yet another significant and impactful step forward for all those living with DEB,” added Brett Kopelan, executive director of debra of America, in a statement. "Enabling caretakers to apply [B-VEC] during their standard-of-care regimen is an enormous positive change, allowing for increased convenience without sacrificing safety. This aspect of the update to the label will only increase the quality of life of those living with this challenging disorder, and that is exactly what our community needs.”¹

Reference

1. Krystal Biotech announces FDA approval of updated Vyjuvek label. Press release. Krystal Biotech Inc. September 15, 2025. Accessed September 15, 2025. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-announces-fda-approval-updated-vyjuvekr-label
2. Fitch J. FDA approves B-VEC to treat dystrophic epidermolysis bullosa patients 6 months and older. Contemporary Pediatrics. May 19, 2025. Accessed September 15, 2025. https://www.contemporarypediatrics.com/view/fda-approves-b-vec-to-treat-dystrophic-epidermolysis-bullosa-patients-6-months-and-older
3. Marinkovich MP, Paller AS, Guide SV, et al. Long-term safety and tolerability of beremagene geperpavec-svdt (B-VEC) in an open-label extension study of patients with dystrophic epidermolysis bullosa. Am J Clin Dermatol. 2025;26(4):623-635. doi:10.1007/s40257-025-00942-y