The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for beremagene geperpavec (B-VEC) for the treatment of patients with dystrophic epidermolysis bullosa.
Dystrophic epidermolysis bullosa (DEB) is a chronic and progressive skin disease caused by mutations in the gene encoding for type VII collagen (COL7), which leads to extremely fragile skin, blistering, and skin loss at the slightest friction. Currently, there is no approved treatment for DEB.
B-VEC is an investigational topical gene therapy designed to deliver 2 copies of the COL7A1 gene when applied directly to DEB wounds. The BLA is supported by data from the double-blind, placebo-controlled phase 3 GEM-3 trial (ClinicalTrials.gov Identifier: NCT04491604) and the phase 2 GEM-1/2 trial (ClinicalTrials.gov Identifier: NCT03536143).
In the GEM-3 trial, 31 patients with DEB provided at least 1 pair of primary target wounds for evaluation, with 1 wound from each pair treated with B-VEC and the other with placebo. Results showed that treatment with B-VEC met both the primary and secondary endpoints demonstrating complete wound healing at 6 months and 3 months, respectively.
In the GEM-1/2 trial, 9 patients with DEB received either B-VEC or placebo for matched wounds over 12 weeks. Treatment with B-VEC was associated with durable wound closure, full-length cutaneous COL7 expression, and anchoring fibril assembly.
As for safety, B-VEC was well tolerated with no drug-related serious adverse events or discontinuations due to treatment.
“We are delighted to receive the FDA’s acceptance of our BLA submission and move one step closer to potentially bringing a medicine to fundamentally treat DEB patients,” said Suma Krishnan, Co-Founder and President, Research & Development of Krystal Biotech. “We are committed to working closely with the FDA to bring this potential, first-ever treatment to patients living with DEB as quickly as possible.”
A Prescription Drug User Fee Act action date of February 17, 2023 has been set for the application.
- FDA accepts Krystal Biotech’s Biologics License Application for dystrophic epidermolysis bullosa. News release. Krystal Biotech, Inc. August 18, 2022. Accessed August 22, 2022. https://www.globenewswire.com/news-release/2022/08/18/2501270/0/en/FDA-Accepts-Krystal-Biotech-s-Biologics-License-Application-for-Dystrophic-Epidermolysis-Bullosa.html
- Krystal Biotech submits Biologics License Application to U.S. FDA seeking approval of B-VEC for the treatment of patients with dystrophic epidermolysis bullosa. News release. Krystal Biotech, Inc. June 22, 2022. Accessed August 22, 2022. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-submits-biologics-license-application-us-fda