CHMP to issue its opinion on DEB gene therapy gel in early 2025
Redosable gel, marketed as Vyjuvek in US, expected to launch next year in EU
An arm of the European Medicines Agency (EMA) is expected to issue its opinion early next year on whether to approve beremagene geperpavec-svdt, which is marketed as Vyjuvek in the U.S., for dystrophic epidermolysis bullosa (DEB).
The Committee for Medicinal Products for Human Use (CHMP) has asked Krystal Biotech, the company marketing the redosable gene therapy gel, to submit written responses to outstanding questions that came about when reviewing the treatment’s marketing authorization application. An oral explanation that was scheduled for Dec. 6 has been cancelled. No major objections remain regarding full approval of the therapy, formerly known as B-VEC, according to the company.
“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 research and development, said in a company press release.
The CHMP reviews data such as how a treatment works, its manufacturing methods, existing efficacy and safety results, and how a company will manage risks related to the drug once it is commercialized in the European Union. During this assessment, the committee may raise questions that need to be answered within an agreed-upon time.
Commercial launch of therapy in Germany planned for 2025
Krystal has stated that all necessary preparations for launching the therapy in Europe are on schedule. With a CHMP opinion expected in the first three months of 2025, a commercial launch is planned for Germany soon after.
DEB is caused by mutations in a gene called COL7A1, which carries instructions for producing type VII collagen (COL7). In a healthy person, this protein helps bind the skin’s layers together. However, when the body cannot produce enough functional COL7, the skin lacks the structural support it needs to stay intact.
For DEB patients, even minor contact, such as rubbing against clothing, can lead to painful blisters, open wounds, and scars.
Beremagene geperpavec-svdt is designed to address the root cause of DEB. The treatment uses a modified, harmless virus to deliver two healthy copies of the COL7A1 gene directly to skin cells in wounds to restore production of functional COL7 protein. This is expected to improve the skin’s structural integrity, help reduce blistering, and resolve wounds.
The gene therapy’s effectiveness and safety were evaluated in comparison to a placebo in the Phase 1/2 GEM-1 trial (NCT03536143) and Phase 3 GEM-3 study (NCT04491604). Nearly all participants in the trials had recessive DEB, a more severe subtype of the disease, which means they have two mutated copies of the COL7A1 gene.
GEM-1 participants applied the gel generally along with bandage changes every two or three days, for about three months. Those in GEM-3 study applied the gel once a week for up to six months.
Results of GEM-1 showed the gel successfully treated all but one chronic, 5-year-old wound. Treated wounds remained healed for at least three months. In contrast, rates of healing and recurring blisters were variable in the placebo group.
In GEM-3, the gene therapy 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%).
The U.S. Food and Drug Administration approved Vyjuvek in May 2023 for treating skin wounds in DEB patients 6 months and older.
