Raj Chovatiya, MD, PhD, on Pediatric Clinical Trials and B-VEC's Milestone Approval in Dermatology

“Hats off to everybody who focuses on [the pediatric] population, when it comes to studies, it is truly not easy. But for genetic diseases that really affect the youngest of the young and can have really bad long-term outcomes, it's really the only way that we can kind of figure out some of these therapies.”

Krystal Biotech’s marketing authorization application for beremagene geperpavec (B-VEC) is up for review in the European Union, following its United States FDA approval in May 2023 for treating dystrophic epidermolysis bullosa (DEB) under the name Vyjuvek.^1,2

B-VEC is unique among gene therapies as it is topically applied in a gel droplet form to the skin and is redosable, with caregivers applying it to wounds on a weekly basis. Rather than using an adeno-associated virus vector, it is a herpes simplex virus type 1-based gene therapy that delivers COL7A1, mutations in which cause DEB, to restore collagen 7 protein, and may have potential to treat other disorders in which COL7A1 is impacted.

CGTLive spoke with Raj Chovatiya, MD, PhD, assistant professor dermatology at the Northwestern University Feinberg School of Medicine, to learn more about the landmark approval of Vyjuvek and what it may mean for the dermatology field. He also discussed some challenges in conducting clinical trials in a pediatric population.

REFERENCES

1. Krystal Biotech announces EMA validation of marketing authorization application for VYJUVEK for the treatment of dystrophic epidermolysis bullosa. News release. Krystal Biotech, Inc. November 27, 2023. Accessed November 27, 2023. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-announces-ema-validation-marketing-authorization
2. Krystal Biotech Receives FDA Approval for the First-Ever Redosable Gene Therapy, VYJUVEK™ (beremagene geperpavec-svdt) for the Treatment of Dystrophic Epidermolysis Bullosa. News release. Krystal Biotech. May 19, 2023. Accessed November 27, 2023. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-receives-fda-approval-first-ever-redosable-gene