Brett Kopelan, MA, and Heather Fullmer on the Potential Impact of Gene Therapy on Epidermolysis Bullosa’s Treatment Landscape

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In observance of Epidermolysis Bullosa Awareness Week, the debra of America representatives discussed unmet needs for the patient population and the approval of the first EB gene therapy.

“...We anticipate [the approval of B-VEC] to be incredibly important. Both Heather and I know some of the people that have been on this therapy for a number of years and they have really seen a huge positive impact on their quality of life: from being wheelchair-bound to no longer being wheelchair-bound and having the time for bandage changes going from 4 hours to a half hour. So, I'm very, very enthused by the impact that this therapeutic agent is going to have on the EB population.”

Epidermolysis bullosa (EB) is a collection of rare connective tissue disorders related to mutations than can occur across 18 different genes. EB is very difficult to manage, with patients being prone to painful skin tears and blisters from common daily activities and having to undergo time-consuming and expensive changes of special bandages. Until recently, the standard of care for EB was largely palliative, but earlier this year, in May, a major breakthrough occurred with the FDA’s approval of Krystal Biotech's beremagene geperpavec (B-VEC, marketed as Vyjuvek), a topical and redosable gene therapy developed by Krystal Biotech, for the treatment of dystrophic epidermolysis bullosa (DEB) in patients 6 months or older.

To kick off EB Awareness Week, observed annually from October 25 to 31 by the patient and clinician community, CGTLive™ interviewed Brett Kopelan, MA, the executive director of the Dystrophic Epidermolysis Bullosa Research Association (debra) of America, and Heather Fullmer, the community engagement director of debra of America, about the current state of care in EB and how the advent of gene therapy could potentially change the treatment landscape for this patient population. Kopelan and Fullmer presented an in-depth picture of the symptoms patients with EB experience and discussed the drawbacks of the current standard of care. Kopelan also gave his view on how B-VEC impacted the experience of patients with DEB who were treated in clinical trials and noted that, although it is very early to draw concrete conclusions, the approval represents a major milestone for the treatment landscape for EB.

REFERENCE
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 October 25, 2023. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-receives-fda-approval-first-ever-redosable-gene

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