In this HCPLive interview, Peter Marinkovich, MD, associate professor of dermatology at Stanford University School of Medicine, spoke about the US Food and Drug Administration (FDA) approval of beremagene geperpavec (B-VEC) (VYJUVEK) for patients with dystrophic epidermolysis bullosa (DEB).¹

Marinkovich had been a primary investigator in the multi-center, randomized, double-blind, placebo-controlled GEM-3 trial sponsored by Krystal Biotech.

The phase 3 trial had met both its primary and secondary endpoints, with B-BEC-treated patients having a substantially greater rate of achieving complete wound healing from DEB than placebo at 3 and 6 months (P <.005).²

Before the PDUFA date’s announcement, trial data on 31 DEB patients indicated that around 70% of wounds had closed within 6 months of treatment, including the patient that had the dominant form of DEB.

“It's just the the culmination of a number of multi-year effort to develop this topical gene therapy drug, and it's so exciting to actually be able to be in this position to be able to offer something back to the very deserving patient population who does not currently have any approved therapies and who are very much in need,” Marinkovich explained.

The treatment’s PDUFA data had been extended to May 2023 resulting from a regulatory update that the FDA deemed important. Krystal Biotech then submitted its manufacturing information update related to a replaced hardware unit in B-VEC’s manufacturing process.

“This is a vector that's based on the HSV-1 backbone,” he said. “HSV-1 over its many thousands of years of cohabitation with humans has evolved a way to be able to evade the immune system…As a gene therapy vector, to have a vector that can evade the immune system, it really makes it much easier to use.”

Marinkovich explained the function of the treatment and how it addresses wound healing for DEB patients. He then went into related research in which he had been involved.

“Some of the things that we're currently studying are the effects of feedback on mucosal surfaces in addition to the skin,” he said. “And that was a very promising report of a single patient who's had their eyes treated, and was able to show some improvement in the vision as well as very good safety over multiple applications.”

Marinkovich noted that these are all areas that EB patients suffer from and that they could benefit from B-VEC therapy. He concluded with his thoughts on the future for DEB patient options.

“I'm super happy about the FDA decision, they also seem to recognize the important need of the EB patients to get a corrective therapy like this one,” Marinkovich said. “And I feel so happy for the EB patients to be able to have corrective therapy and be able to finally, after all these years, be able to actually start to get some benefit.”

The quotes contained here were edited for clarity. For further information about Marinkovich’s take on the FDA’s approval of B-VEC therapy, view the video above.

References

1. ^{Smith T. FDA Approves B-VEC Topical Gene Therapy for Dystrophic Epidermolysis Bullosa. HCPLive. May 19, 2023. https://www.hcplive.com/view/fda-bvec-topical-gene-therapy-dystrophic-epidermolysis-bullosa}
2. ^{Kunzumann K. Topical Gene Therapy B-VEC Reaches Phase 3 Efficacy Endpoints for DEB Disease Reversal. March 26, 2022. Accessed May 19, 2023. https://www.hcplive.com/view/topical-gene-therapy-b-vec-phase-3-efficacy-endpoints-deb-disease-reversal.}