| Clinical data | |
|---|---|
| Trade names | Vyjuvek |
| Other names | KB-103 |
| License data | |
| Routes of administration | Topical |
| ATC code | |
| Legal status | |
| Legal status | |
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Beremagene geperpavec, sold under the brand name Vyjuvek, is a gene therapy for the treatment of wounds.[1][3] Beremagene geperpavec is the first approved gene therapy to use herpes-simplex virus type 1 as a vector.[4][3] Beremagene geperpavec is a genetically modified (engineered in a laboratory) herpes-simplex virus used to deliver normal copies of the COL7A1 gene to the wounds.[3]
The most common adverse reactions include itching, chills, redness, rash, cough and runny nose.[3]
Beremagene geperpavec was approved for medical use in the United States in May 2023.[2][3]
Medical uses
Beremagene geperpavec is indicated for the treatment of wounds in people with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.[1][2][3]
History
The safety and effectiveness of beremagene geperpavec was established primarily in a randomized, double-blinded, placebo-controlled study involving a total of 31 subjects with dystrophic epidermolysis bullosa, including 30 subjects with recessive dystrophic epidermolysis bullosa and one subject with dominant dystrophic epidermolysis bullosa. In the study, two dystrophic epidermolysis bullosa wounds of comparable size on each participant were identified and randomized to receive either topical administration of beremagene geperpavec or the placebo on a weekly basis.[3] The age of the subjects ranged from 1 year to 44 years (mean age 17 years).[3] Efficacy was established by improved wound healing, defined as the difference in the proportion of confirmed complete (100%) wound closure between the beremagene geperpavec-treated and the placebo-treated wounds at 24 weeks.[3] Sixty-five percent of the beremagene geperpavec-treated wounds completely closed while only 26% of the placebo-treated wound completely closed.[3]
The U.S. Food and Drug Administration (FDA) granted the application for beremagene geperpavec orphan drug, fast track, regenerative medicine advanced therapy, and priority review designations along with a rare pediatric disease priority review voucher.[3] The FDA granted the approval of Vyjuvek to Krystal Biotech, Inc.[3]
References
- 1 2 3 "Vyjuvek kit". DailyMed. 19 May 2023. Archived from the original on 21 June 2023. Retrieved 20 June 2023.
- 1 2 3 "Vyjuvek". U.S. Food and Drug Administration (FDA). 19 May 2023. 125774. Archived from the original on 24 May 2023. Retrieved 30 May 2023.
This article incorporates text from this source, which is in the public domain. - 1 2 3 4 5 6 7 8 9 10 11 12 "FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa". U.S. Food and Drug Administration (FDA) (Press release). 19 May 2023. Archived from the original on 27 May 2023. Retrieved 28 May 2023. This article incorporates text from this source, which is in the public domain.
- ↑ "First herpesvirus gene therapy". Nature Biotechnology. 41 (6): 739. June 2023. doi:10.1038/s41587-023-01835-3. PMID 37316736. S2CID 259157518. Archived from the original on 20 June 2023. Retrieved 19 June 2023.
This article incorporates public domain material from the United States Department of Health and Human Services
Further reading
- Guide SV, Gonzalez ME, Bağcı IS, Agostini B, Chen H, Feeney G, et al. (December 2022). "Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa". The New England Journal of Medicine. 387 (24): 2211–2219. doi:10.1056/NEJMoa2206663. PMID 36516090. S2CID 254728349.
- Gurevich I, Agarwal P, Zhang P, Dolorito JA, Oliver S, Liu H, et al. (April 2022). "In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial". Nature Medicine. 28 (4): 780–788. doi:10.1038/s41591-022-01737-y. PMC 9018416. PMID 35347281.