Krystal Biotech's B-VEC for Dystrophic Epidermolysis Bullosa Gets Expanded FDA Label

Today, Krystal Biotech announced a new label update has been approved by the US Food and Drug Administration (FDA) for beremagenegeperpavec-svdt (B-VEC/YJUVEK).¹ The update expands B-VEC's eligible patient population to include dystrophic epidermolysis bullosa (DEB) patients from birth. Patients and caregivers can now apply the medication and manage wound dressings at home on their own. Additionally, wound dressings are now permitted to be removed as part of the next dressing change rather than waiting 24 hours.

“We believe these changes further reinforce VYJUVEK’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. “Moreover, by providing patients and their caregivers the ability to apply VYJUVEK themselves, we have made it easier for patients to integrate VYJUVEK into their daily routines and lifestyle. Overall, we believe that these advancements solidify VYJUVEK as the standard of care for all DEB wounds, regardless of wound size and severity, and will further improve compliance.”¹

This label update is supported by new research, including real-world data following B-VEC's launch in the US and an open-label extension trial published in the American Journal of Clinical Dermatology (NCT04917874). In Marinkovich et al., high levels of patient satisfaction and treatment response were recorded in participants who continued the therapy beyond 6 months. Wounds that received B-VEC during phase 3, including when applied by patients and caregivers, maintained high closure rates during the open-label extension (61.1–89.5%).² No new safety signals were detected. The most common treatment-emergent adverse events (incidence >5%) are itching, chills, redness, rash, cough, and runny nose.

“The updates to the VYJUVEK label are yet another significant and impactful step forward for all those living with DEB,” said Brett Kopelan, Executive Director of Debra of America. “Enabling caretakers to apply VYJUVEK during their standard of care regimen is an enormous positive change, allowing for increased convenience without sacrificing safety. The Krystal team has always prioritized patient safety and convenience when it comes to the use of VYJUVEK and them advocating for these updates is not surprising given Krystal’s patient-centric approach. 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

B-VEC was approved by the FDA for the treatment of DEB in May 2023. The approval was based on positive data from 2 placebo-controlled clinical trials, GEM 3 (NCT04491604) and GEM1/2 (NCT03536143). B-VEC was granted priority review designation in August 2022, as DEB is a rare and severe blistering disorder that results from a mutation in gene encoding type VII collagen (COL7A1). Repeated cycles of blistering can result in scarring, malignancy, infection, limb loss, and early death.

B-VEC is a non-invasive, topical, redosable gene therapy created to administer 2 copies of the COL7A1 gene when directly applied to DEB wounds. B-VEC treats DEB at the molecular level by giving patients’ cells a template to produce normal COL7 proteins. It is currently approved in the US, Europe, and Japan, and is the first FDA-approved drug for DEB in the US.

References

1. Krystal Biotech Announces FDA Approval of Updated VYJUVEK® Label. News release. Krystal Biotech. Published 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. Marinkovich MP, Paller AS, Guide SV, et al. Long-Term Safety and Tolerability of BeremageneGeperpavec-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