FDA Approves Beremagene Geperpavec-svdt (Vyjuvek) Label Update in DEB

Krystal Biotech has announced the United States Food and Drug Administration (FDA) approval of a label update for beremagene geperpavec-svdt (Vyjuvek) that expands the treatment eligible patient population to include dystrophic epidermolysis bullosa (DEB) patients from birth and provides patients full flexibility with respect to beremagene geperpavec-svdt application and managing wound dressings.1

According to a September 15, 2025, press release from the Company, in addition to allowing patients with DEB and their caregivers to apply beremagene geperpavec-svdt at home on their own, the label update also affords patients greater flexibility in managing wound dressings, as they are now permitted to be removed as part of the next dressing change rather than waiting 24 hours.1

“The updates to the [beremagene geperpavec-svdt] label are yet another significant and impactful step forward for all those living with DEB” Brett Kopelan, Executive Director of DEBRA of America, said in a statement.1 “Enabling caretakers to apply [beremagene geperpavec-svdt] 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 [beremagene geperpavec-svdt] 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.”

A non-invasive, topical, redosable genetic medicine, beremagene geperpavec-svdt is designed to deliver 2 copies of the COL7A1 gene when applied directly to DEB wounds. It was designed to treat DEB at the molecular level by providing the patient’s skin cells the template to make normal COL7 protein, thereby addressing the fundamental disease-causing mechanism. Beremagene geperpavec-svdt is approved in the United States, Europe, and Japan.11,2

The FDA approved beremagene geperpavec-svdt for the treatment of DEB in 2023, making it the first-ever redosable gene therapy and the first and only medicine approved by the FDA for the treatment of DEB, both recessive and dominant, that can be administered by a healthcare professional in either a healthcare professional setting or in the home.2

The decision was based on data from 2 clinical studies, GEM-1/2 and GEM-3. GEM-1/2 was an intra-patient, open label, single center, randomized, placebo-controlled study showing that repeat topical applications of beremagene geperpavec-svdt were associated with durable wound closure, full-length cutaneous COL7 expression, and anchoring fibril assembly with minimal reported adverse events.2

The GEM-3 trial was an intra-patient, double-blinded, multi-center, randomized, placebo-controlled study that met both its primary endpoint of complete wound healing at 6 months and its key secondary endpoint of complete wound healing at 3 months. Beremagene geperpavec-svdt was well tolerated with no drug-related serious adverse events or discontinuations due to treatment-related events.2

The September 15, 2025, label update is based on real-world data collected since beremagene geperpavec-svdt launched in the United States as well as results from the open label extension study conducted in the United States, reinforcing the long-term safety and efficacy of beremagene geperpavec-svdt across patients of all ages, including in cases of patient or caregiver application.1

“We believe these changes further reinforce [beremagene geperpavec-svdt]’s leadership position as the most flexible, convenient, and disease modifying medicine to treat DEB patients in the United States,” said Krish Krishnan, Chairman and CEO of Krystal Biotech.1 “Moreover, by providing patients and their caregivers the ability to apply [beremagene geperpavec-svdt] themselves, we have made it easier for patients to integrate [beremagene geperpavec-svdt] into their daily routines and lifestyle. Overall, we believe that these advancements solidify [beremagene geperpavec-svdt] as the standard of care for all DEB wounds, regardless of wound size and severity, and will further improve compliance.”

References

1. Krystal Biotech. Krystal Biotech Announces FDA Approval of Updated VYJUVEK® Label. 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. Smith T. FDA Approves B-VEC Topical Gene Therapy for Dystrophic Epidermolysis Bullosa. HCPLive. May 19, 2023. Accessed September 15, 2025. https://www.hcplive.com/view/fda-bvec-topical-gene-therapy-dystrophic-epidermolysis-bullosa