Krystal Biotech’s marketing authorization application (MAA) for beremagene geperpavec (B-VEC, marketed as Vyjuvek in the United States), an investigational gene therapy intended to treat dystrophic epidermolysis bullosa (DEB), has been validated by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).1
CHMP is expected to give an opinion on the MAA in the second half of next year. The validation is the latest in a series of positive developments for B-VEC's future in the European Union. Just a few months ago, in September 2023, a positive opinion on the Pediatric Investigation Plan for B-VEC was issued by the EMA Pediatric Committee. Pending marketing approval of B-VEC in the EU, this opinion would add an additional 2 years of marketing exclusivity to the 10 years of marketing exclusivity already expected upon approval in the EU. The EMA has also previously granted B-VEC both orphan drug and priority medicines designations.
“The validation of our MAA for review by CHMP is an important step toward our goal to bring VYJUVEK to patients in the EU who are living with DEB,” Suma Krishnan, MS, MBA, the president of research & development at Krystal Biotech, said in a statement.1 “We look forward to working closely with EMA through the MAA review process, as Vyjuvek has the potential to fulfill an unmet medical need for DEB patients and their families.”
B-VEC is redosable and applied topically directly to DEB wounds. It is intended to deliver 2 functional copies of the disease-targeted gene, COL7A1, via a herpes-simplex virus type 1 vector, to allow the skin cells to produce the normal COL7 protein. The gene therapy was approved in the US by the FDA in May 2023; it was the first gene therapy to be approved for a dermatological condition.2 The indication covers use in patients with DEB who are 6 months of age or older. B-VEC’s biologics license application in the US was supported by data from 2 intrapatient, placebo-controlled clinical trials: the phase 2 GEM-1/2 (NCT03536143) study and the phase 3 GEM-3 study (NCT04491604). Six months out from the approval, CGTLive™ spoke with several experts in the field to learn more about B-VEC's effect on the treatment landscape for DEB.
- Krystal Biotech's marketing authorization application (MAA) for B-VEC, a gene therapy for dystrophic epidermolysis bullosa (DEB), has been validated by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), marking a crucial step in the approval process.
- The MAA validation follows prior grants of orphan drug and priority medicines designations.
- B-VEC, already approved in the US, is a redosable gene therapy applied topically to DEB wounds.
“I've been prescribing it to a number of patients, and they have been really enjoying its use and are seeing healing of their skin...,” M. Peter Marinkovich, MD, an associate professor of dermatology at Stanford University, and an investigator on the GEM trials, told CGTLive. “Up until now, EB patients had no corrective therapy at all. It was frustrating for the patients, families, and physicians alike. We were able to use fancy wound dressings, fight infection, and try to promote nutrition, but we just helplessly watched the blisters form and couldn’t do anything about it... They've all been waiting for such a long time for this. It's just so exciting to get a corrective therapy to them.”
In observance of EB Awareness Week, which is held each year from October 25 to 31, CGTLive also recently spoke with Brett Kopelan, MA, the executive director of the Dystrophic Epidermolysis Bullosa Research Association (debra) of America, about the rapidly changing landscape of care for EB and the options that may become available for patients with EB in the future. Kopelan noted that with several other therapeutics for EB currently in development, B-VEC may eventually become part of a multitherapeutic approach to treating EB. Specifically, he discussed Abeona Therapeutics’ EB-101, an investigational autologous cell therapy intended to treat recessive DEB; Castle Creek Biosciences’ dabocemagene autoficel (FCX-007, D-Fi), an investigational autologous ex vivo fibroblast therapeutic agent delivered by intradermal injections; and a company in Germany that is working on a systemic cell therapy approach to treating EB.3,4
1. Krystal Biotech announces EMA validation of marketing authorization application for VYJUVEK for the treatment of dystrophic epidermolysis bullosa. News release. Krystal Biotech, Inc. November 27, 2023. Accessed November 27, 2023. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-announces-ema-validation-marketing-authorization
2. Krystal Biotech Receives FDA Approval for the First-Ever Redosable Gene Therapy, VYJUVEK™ (beremagene geperpavec-svdt) for the Treatment of Dystrophic Epidermolysis Bullosa. News release. Krystal Biotech. May 19, 2023. Accessed November 27, 2023. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-receives-fda-approval-first-ever-redosable-gene
3. Abeona Therapeutics submits biologics license application to U.S. FDA seeking priority review and approval of EB-101 for the treatment of patients with Recessive Dystrophic Epidermolysis Bullosa. News release. Abeona Therapeutics. September 26, 2023. Accessed November 27, 2023. https://investors.abeonatherapeutics.com/press-releases/detail/265/abeona-therapeutics-submits-biologics-license-application
4. Castle Creek Biosciences acquires Novavita Thera to expand innovative cell and gene therapy platform. News release. Castle Creek Biosciences. January 10, 2022. Accessed November 27, 2023. https://castlecreekbio.com/castle-creek-biosciences-acquires-novavita-thera-to-expand-innovative-cell-and-gene-therapy-platform/