The European Medicines Agency (EMA) has postponed its decision regarding the marketing authorization application for beremagene geperpavec-svdt (B-VEC), a gene therapy developed by Krystal Biotech for the treatment of dystrophic epidermolysis bullosa (DEB). The EMA's Committee for Medicinal Products for Human Use (CHMP) has requested that Krystal Biotech submit further written responses to address remaining issues, leading to the cancellation of the previously scheduled oral explanation.
Regulatory Update
Krystal Biotech announced that the CHMP cancelled the oral explanation, which was initially set for December 6, 2024. Instead, the company will provide written responses to the outstanding issues. Despite this delay, Krystal Biotech remains optimistic, stating that there are no major objections outstanding from the EMA regarding the full approval of B-VEC.
Suma Krishnan, President of Research and Development at Krystal Biotech, expressed confidence in the company's ability to address the remaining post-marketing issues. "We are confident in our ability to address the remaining post-marketing issues, and we believe that this additional exchange with EMA will ultimately maximize benefit and convenience to patients suffering from DEB," Krishnan said.
The company now anticipates receiving a CHMP opinion in the first quarter of 2025. The planned commercial launch in Germany is still slated for the second quarter of 2025, indicating minimal disruption to Krystal Biotech's European market entry strategy.
About Dystrophic Epidermolysis Bullosa (DEB)
DEB is a rare and severe genetic disorder characterized by extremely fragile skin and mucosal tissues. The condition arises from mutations in the COL7A1 gene, which is responsible for producing type VII collagen (COL7). This protein is essential for forming anchoring fibrils that connect the dermis and epidermis. The absence of functional anchoring fibrils in DEB patients results in skin that blisters and tears easily from minor friction or trauma.
Patients with DEB often suffer from chronic open wounds, leading to skin infections, fibrosis, and an increased risk of squamous cell carcinoma, which can be fatal in severe cases. The disease significantly impacts the quality of life for affected individuals.
B-VEC: A Novel Gene Therapy Approach
B-VEC is a non-invasive, redosable gene therapy designed to deliver two copies of the COL7A1 gene directly to the patient's cells. This approach aims to treat DEB at the molecular level by providing the necessary template for cells to produce normal COL7 protein, thereby addressing the underlying cause of the disease. In May 2023, the U.S. Food and Drug Administration (FDA) approved B-VEC, which is marketed in the U.S. under the brand name VYJUVEK®.
The efficacy and safety of beremagene geperpavec-svdt were demonstrated in the Phase 1/2 GEM-1 trial (NCT03536143) and the Phase 3 GEM-3 study (NCT04491604). The GEM-3 study, for instance, showed that B-VEC was associated with a significantly higher proportion of completely healed wounds relative to the placebo gel at both three months (71% vs. 20%) and six months (67% vs. 22%).
