NEW YORK – Krystal Biotech expects to receive an opinion on its gene therapy for dystrophic epidermolysis bullosa (DEB) from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) in Q1 2025, the company said Monday.
The European Commission considers CHMP opinions in deciding whether to grant marketing authorization for drug products in the EU.
Krystal's gene therapy, beremagene geperpavec, or B-VEC, was approved last year by the US Food and Drug Administration, where it is sold under the brand name Vyjuvek. The gene therapy uses a genetically modified herpes-simplex virus type 1 vector to deliver two functional copies of COL7A1 to patients. Mutations in COL7A1 cause DEB, a serious skin condition in which patients have fragile skin that easily blisters and tears. Krystal's treatment is in the form of a topical gel that a healthcare professional applies to patients' wounds weekly.
The EMA had scheduled a Dec. 6 meeting, during which Krystal was slated to present information before a committee and answer questions about the gene therapy and its marketing authorization application. However, on Tuesday, Krystal said the EMA canceled that meeting and requested the firm submit additional written information.
There are no major objections outstanding from the EMA in terms of approval for the gene therapy, Krystal said, and it continues to expect to launch the product in Germany in Q2 2025.
"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," Suma Krishnan, Krystal's president of R&D, said in a statement.
Separately, Pittsburgh-based Krystal is developing an eyedrop version of the gene therapy as a potential treatment for ocular complications of DEB.