M. Peter Marinkovich, MD, on Bringing RDEB Treatment to the Local Level With B-VEC


The associate professor of dermatology at Stanford University discussed how the May 2023 approval of B-VEC may shift the treatment field for RDEB.

“Up until now, most of the care for EB patients has been confined to these tertiary specialized centers, referral centers. That's because in the local communities, there wasn't much that the local dermatologists and pediatricians could do to help the EB patients. But now that's changed. Now with the therapy, they can make a big impact on the patient's lives. So, I would predict that this therapy is going to get more and more pediatricians and dermatologists actually seeing EB patients in the local community, which could be a really good thing.”

It’s been almost 1 year since Krystal Biotech’s beremagene geperpavec (B-VEC) became the first approved gene therapy in the dermatology field in May 2023.The therapy, marked at as Vyjuvek, is indicated for treating dystrophic epidermolysis bullosa (DEB). CGTLive spoke with some experts in the field involved in its development to look back at the approval, how it is affecting the dermatology field, and their future outlook in the field.

In this clip, CGTLive spoke with M. Peter Marinkovich, MD, an associate professor of dermatology at Stanford University, and an investigator on the pivotal GEM trials that evaluated B-VEC. Marinkovich discussed the impact of B-VEC's new place in the treatment landscape on the dermatology field and also the gene therapy field in general, as it represents the first redosable, topical gene therapy approval. He predicted that the treatment of patients with RDEB may shift more to local communities considering the ease of treatment with B-VEC and also that younger patients with RDEB will more easily be treated as well.

FDA approves first topical gene therapy for treatment of wounds in patients with dystrophic epidermolysis bullosa. News release. FDA. May 19, 2023. Accessed May 19, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-topical-gene-therapy-treatment-wounds-patients-dystrophic-epidermolysis-bullosa
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