Beremagene geperpavec explained

Tradename:	Vyjuvek
Dailymedid:	Beremagene geperpavec
Routes Of Administration:	Topical
Atc Prefix:	D03
Atc Suffix:	AX16
Legal Us:	Rx-only
Legal Us Comment:	^[1]
Cas Number:	2241888-62-8
Drugbank:	DB17831
Unii:	AQN7K24KQU
Kegg:	D12632
Synonyms:	KB-103

Beremagene geperpavec, sold under the brand name Vyjuvek, is a gene therapy for the treatment of wounds.^[2] Beremagene geperpavec is the first approved gene therapy to use herpes-simplex virus type 1 as a vector.^[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.

The most common adverse reactions include itching, chills, redness, rash, cough and runny nose.

Beremagene geperpavec was approved for medical use in the United States in May 2023.

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.^[4]

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. The age of the subjects ranged from 1 year to 44 years (mean age 17 years). 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. Sixty-five percent of the beremagene geperpavec-treated wounds completely closed while only 26% of the placebo-treated wound completely closed.

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. The FDA granted the approval of Vyjuvek to Krystal Biotech, Inc.

Further reading

• Guide SV, Gonzalez ME, Bağcı IS, Agostini B, Chen H, Feeney G, Steimer M, Kapadia B, Sridhar K, Quesada Sanchez L, Gonzalez F, Van Ligten M, Parry TJ, Chitra S, Kammerman LA, Krishnan S, Marinkovich MP . 6 . Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa . The New England Journal of Medicine . 387 . 24 . 2211–2219 . December 2022 . 36516090 . 10.1056/NEJMoa2206663 . 254728349 . free .
• Gurevich I, Agarwal P, Zhang P, Dolorito JA, Oliver S, Liu H, Reitze N, Sarma N, Bagci IS, Sridhar K, Kakarla V, Yenamandra VK, O'Malley M, Prisco M, Tufa SF, Keene DR, South AP, Krishnan SM, Marinkovich MP . 6 . In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial . Nature Medicine . 28 . 4 . 780–788 . April 2022 . 35347281 . 9018416 . 10.1038/s41591-022-01737-y .

Notes and References

1. Web site: Vyjuvek kit . DailyMed . 19 May 2023 . 20 June 2023 . 21 June 2023 . https://web.archive.org/web/20230621044100/https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fe207c8c-04b4-4fdf-87cf-6dbc7eb6e9c2 . live .
2. FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa . U.S. Food and Drug Administration (FDA) . 19 May 2023 . 28 May 2023 . 27 May 2023 . https://web.archive.org/web/20230527205551/https://www.fda.gov/news-events/press-announcements/fda-approves-first-topical-gene-therapy-treatment-wounds-patients-dystrophic-epidermolysis-bullosa . live .
3. June 2023 . First herpesvirus gene therapy . . 41 . 6 . 739 . 10.1038/s41587-023-01835-3 . 37316736 . 259157518 . 19 June 2023 . 20 June 2023 . https://web.archive.org/web/20230620021433/https://www.nature.com/articles/s41587-023-01835-3 . live .
4. Web site: Vyjuvek . U.S. Food and Drug Administration (FDA) . 19 May 2023 . 30 May 2023 . 125774 . 24 May 2023 . https://web.archive.org/web/20230524115753/https://www.fda.gov/vaccines-blood-biologics/vyjuvek . live .