beremagene geperpavec
{{Short description|Gene therapy}}
{{Use American English|date=May 2023}}
{{Use dmy dates|date=May 2023}}
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| tradename = Vyjuvek
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| DailyMedID = Beremagene geperpavec
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| routes_of_administration = Topical
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| ATC_prefix = D03
| ATC_suffix = AX16
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| legal_US = Rx-only
| legal_EU = Rx-only
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| CAS_number = 2241888-62-8
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| DrugBank = DB17831
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| UNII = AQN7K24KQU
| KEGG = D12632
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| synonyms = KB-103, beremagene geperpavec-svdt
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Beremagene geperpavec, sold under the brand name Vyjuvek, is a gene therapy used for the treatment of wounds.{{cite press release | title=FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa | website=U.S. Food and Drug Administration (FDA) | date=19 May 2023 | url=https://www.fda.gov/news-events/press-announcements/fda-approves-first-topical-gene-therapy-treatment-wounds-patients-dystrophic-epidermolysis-bullosa | access-date=28 May 2023 | archive-date=27 May 2023 | archive-url=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 | url-status=live }} {{PD-notice}} Beremagene geperpavec is the first approved gene therapy to use herpes-simplex virus type 1 as a vector.{{cite journal |date=June 2023 |title=First herpesvirus gene therapy |url=https://www.nature.com/articles/s41587-023-01835-3 |journal=Nature Biotechnology |volume=41 |issue=6 |pages=739 |doi=10.1038/s41587-023-01835-3 |pmid=37316736 |s2cid=259157518 |access-date=19 June 2023 |archive-date=20 June 2023 |archive-url=https://web.archive.org/web/20230620021433/https://www.nature.com/articles/s41587-023-01835-3 |url-status=live |url-access=subscription }} 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, and in the European Union in April 2025.
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.{{cite web | title=Vyjuvek | website=U.S. Food and Drug Administration (FDA) | date=19 May 2023 | url=https://www.fda.gov/vaccines-blood-biologics/vyjuvek | access-date=30 May 2023 | id=125774 | archive-date=24 May 2023 | archive-url=https://web.archive.org/web/20230524115753/https://www.fda.gov/vaccines-blood-biologics/vyjuvek | url-status=dead }} {{PD-notice}}
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 US 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.
Society and culture
= Legal status =
In February 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Vyjuvek, intended for the topical treatment of wounds in people with dystrophic epidermolysis bullosa caused by mutations in the collagen type 7 alpha 1 chain (COL7A1) gene. As Vyjuvek is an advanced therapy medicinal product, the CHMP positive opinion is based on an assessment by the Committee for Advanced Therapies. The applicant for this medicinal product is Krystal Biotech Netherlands, B.V.{{cite web | title=Vyjuvek EPAR | website=European Medicines Agency (EMA) | date=27 February 2025 | url=https://www.ema.europa.eu/en/medicines/human/EPAR/vyjuvek | access-date=4 March 2025}} Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. Vyjuvek was authorized for medical use in the European Union in April 2025.{{cite web | title=Vyjuvek PI | website=Union Register of medicinal products | date=24 April 2025 | url=https://ec.europa.eu/health/documents/community-register/html/h1918.htm | access-date=3 May 2025}}
References
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{{HHS content|url=https://www.fda.gov/}}
Further reading
- {{cite journal | vauthors = 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 | display-authors = 6 | title = Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa | journal = The New England Journal of Medicine | volume = 387 | issue = 24 | pages = 2211–2219 | date = December 2022 | pmid = 36516090 | doi = 10.1056/NEJMoa2206663 | s2cid = 254728349 | doi-access = free }}
- {{cite journal | vauthors = 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 | display-authors = 6 | title = In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial | journal = Nature Medicine | volume = 28 | issue = 4 | pages = 780–788 | date = April 2022 | pmid = 35347281 | pmc = 9018416 | doi = 10.1038/s41591-022-01737-y }}
{{Preparations for treatment of wounds and ulcers}}
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