European Medicines Agency recommends ‘landmark’ gene therapy for epidermolysis bullosa

Key takeaways:

• The recommendation was extended to Vyjuvek, a topical that delivers gene copies that address the root cause of the disease.
• A final decision on Vyjuvek’s approval is expected in the second quarter of 2025.

Editor’s note: This is a developing news story. Please check back soon for updates.

The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended the approval of Vyjuvek for the treatment of wounds in patients with dystrophic epidermolysis bullosa, Krystal Biotech announced in a press release.

The Committee’s positive opinion includes support for the administration of Vyjuvek (beremagene geperpavec-svdt, Krystal Biotech), a noninvasive, topical gene therapy that is also known as B-VEC, both in-office and at home for dystrophic epidermolysis bullosa (DEB).

A final decision is expected in the second quarter of 2025, which will apply to all European Union member states and Iceland, Norway and Liechtenstein, according to the release.

“We are excited to be able to provide DEB patients with the first treatment that corrects the genetic defect and makes a true difference in their lives,” Cristina Has, MD, professor and head of the genodermatoses clinic in the department of dermatology at the University of Freiburg in Germany, said in the release. “By addressing the very first stage in the complex pathophysiology of DEB, Vyjuvek is a landmark. It is amazing how simple and noninvasive its use is, even in infants.”

DEB is a rare, inherited genetic disease caused by mutations in the COL7A1 gene that create blistering on the skin. When applied to DEB wounds, Vyjuvek reportedly delivers two copies of the COL7A1 gene to help the skin produce normal collagen type VII protein and address the root cause of the disease.

The Committee’s positive opinion of Vyjuvek was supported by “a comprehensive clinical dataset,” which included results from Krystal Biotech’s phase 1/2 GEM-1 and phase 3 GEM-3 studies.

If approved, the technology will be under the registered European trademark, Vyjuvek, according to the release.