How FDA-approved B-VEC will help children with dystrophic epidermolysis bullosa

Transcript (Edited for clarity)

Contemporary Pediatrics®
Hi, and thanks so much for visiting Contemporary Pediatrics. I'm editor Joshua Fitch.

Bernard Cohen, MD
It's a pleasure to be here. I'm in the transitional retirement program at Johns Hopkins. I trained in pediatrics and dermatology and I certainly have a special interest focused in children with dermatologic disorders.

Fitch
Dr. Cohen, thank you so much for joining us. Today we're discussing the FDA approval of crystal biotechs B-VEC. for the treatment of dystrophic, epidermolysis bullosa, or DEB, in patients 6 months and older. Dr. Cohen, how can this treatment option for DEB patients affect the overall treatment landscape? And what will frankly be the immediate benefit?

Cohen
Well, the studies that have been done over the last couple of years, and the most recent one was, you know, phase 3, looking for approval, it's a couple things. One is that it looks to be very safe, and very effective. It works by application to open blistered, or ulcerated lesions. So it doesn't fully solve all the issues because it'd be nice to fix these patients from head to toe, even in areas that are not active at the time. But again, it does facilitate really rapid, relatively rapid, and persistent healing of blisters in areas that are particularly predisposed to trauma where a lot of these ulcerations occur, because those are places like elbows and knees and places like that, that have the most trauma. Those are the areas that get the most ulcerated lesions and can be very uncomfortable.

Contemporary Pediatrics
Thank you, Dr. Cohen, how can this treatment option compare to others currently approved for the age range of 6 months and older? Or is this something relatively new that could greatly change the landscape for treatment in DEB patients.

Cohen
Some of the other options require general anesthesia for extended periods of time. They may also be quite painful and uncomfortable. And again, question of their persistence long term is also come under question.

Contemporary Pediatrics®
You mentioned in the phase 3 clinical trials, this seemed relatively safe, can you just speak to its safety and efficacy going forward now that this drug has been FDA approved?

Cohen
This topical therapy, again, it's effective only in areas where there's an ulceration. So again, it's not something that can be used all over the body; those are areas that probably tend to have recurrent lesions. And it's a bit more of a problem in those areas. And it responds well, it is painless, it is applied topically. And from a safety standpoint, although there is a very, very small risk of some redness and some irritation. Other than other than that I'm not aware of any serious side effects associated with this medication. The other thing is that it's not something that produces permanent resolution, even in the areas that are all sorted, where it's applied. It does require continued long term application or reapplication.

Contemporary Pediatrics
With a weekly administration process, could it be an easier treatment choice for patients compared to other options on the market? I mean, it certainly sounds less invasive.

Cohen
I mean, yes, it's less invasive, it's less destructive, it's really pretty much painless. Other than that, it's otherwise sort of risk free. I mean, when people hear about the transport device being herpes simplex type 1, what they don't realize is that has been modified in such a way that it cannot reproduce in the skin. So it doesn't pose a risk of patients acquiring herpes from the topical application. So it's very safe. I mean, ideally, it would be wonderful if this could be applied, you know, head to toe, and you could get genetic improvement on all sides. But right now, again, from a safety standpoint, pain standpoint, from a complication standpoint, it's really virtually risk free, and very safe.

Contemporary Pediatrics®
Thank you, Dr. Cohen, what else if anything, would you like to add regarding this FDA approval going forward?

Cohen
Well, again, I think from a safety standpoint, it's, one of the safest things out there in terms of therapy. And what people don't realize is that they're not only a risk in childhood, but as these kids go on to become adults, the risk of skin cancer goes up significantly. And the skin cancer is probably a greatest risk in areas where the erosions and ulcerations appear, so that by shutting down the formation of these erosions and ulcerations, my hope would be is that long term, it would reduce the risk of them developing squamous cell carcinoma, which can be dangerous in any population, but particularly this population, because that's a long term risk. So not just short term risks, in terms of reduction of pain and healing of the ulcers, but also reduction of the long term risk as well.

Reference

FDA approves first topical gene therapy for treatment of wounds in patients with dystrophic epidermolysis bullosa. 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