Identification
- Summary
Beremagene geperpavec is gene therapy used to treat wounds associated with dystrophic epidermolysis bullosa.
- Brand Names
- Vyjuvek
- Generic Name
- Beremagene geperpavec
- DrugBank Accession Number
- DB17831
- Background
Beremagene geperpavec is a live, replication-defective herpes simplex virus type 1 (HSV-1)-based vector therapy. Developed by Krystal Biotech, it was first approved by the FDA on May 19, 2023, for the treatment of wounds associated with dystrophic epidermolysis bullosa (DEB).5 DEB is caused by mutations in the COL7A1 gene that encodes collagen VII (COL7), a major component of the anchoring fibrils for dermal–epidermal cohesion.1 Beremagene geperpavec is genetically modified to deliver COL7A1, thereby restoring the levels of the COL7 protein.4
- Type
- Biotech
- Groups
- Approved
- Biologic Classification
- Gene Therapies
Other gene therapies - Synonyms
- B-VEC
- Bercolagene telserpavec
- Dna (recombinant herpes simplex virus type 1 strain kos plasmid vector kb103 human collagen col7a1-specifying)
- External IDs
- KB-103
- KB103
Pharmacology
- Indication
Beremagene geperpavec is indicated for the treatment of wounds in patients six months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.4
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Indication Type Indication Combined Product Details Approval Level Age Group Patient Characteristics Dose Form Treatment of Wound •••••••••••• - Contraindications & Blackbox Warnings
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Beremagene geperpavec is a gene therapy that restores the deficient levels of human type VII collagen (COL7) protein.4 In RDEB mice and human RDEB xenografts, beremagene geperpavec restored C7 expression in keratinocytes and fibroblasts.1 In a trial comprising patients with dystrophic epidermolysis bullosa, the topical administration of beremagene geperpavec was associated with greater wound healing at three and six months compared to placebo.3
- Mechanism of action
Collagen VII (C7, COL7), encoded by the COL7A1 gene, is an anchoring fibril component that holds the epidermis and dermis together to maintain skin integrity.1,2 COL7 is synthesized by epidermal keratinocytes and dermal fibroblasts.2 Dystrophic epidermolysis bullosa (DEB) is an inherited disorder caused by COL7A1 gene mutations, leading to reduced or deficient levels of biologically active and functional COL7. Deficient levels of COL7 result in no functional anchoring fibrils and impaired dermal-epidermal adhesion.1,2 DEB is associated with blistering, wounding, and scarring of the skin and other organs starting at birth.1,4
Beremagene geperpavec is a herpes simplex virus type 1 (HSV-1)-based vector genetically modified to express COL7. Upon topical application to wounds, beremagene geperpavec enters both keratinocytes and fibroblasts and the vector genome is deposited in the nucleus. Once in the nucleus, transcription of the encoded human COL7A1 is initiated to produce and secrete COL7 by the cell in its mature form. COL7 molecules arrange themselves into long, thin bundles that form anchoring fibrils.4
- Absorption
There is limited information available regarding the absorption of beremagene geperpavec. In an initial clinical study, viral vector DNA was detected in skin swab samples in all nine treated subjects, with a maximum level ranging from 5.1×104 to 4.1×108 vector genomes.4
- Volume of distribution
There is no information available.
- Protein binding
There is no information available.
- Metabolism
There is no information available.
- Route of elimination
There is limited information available. In an initial clinical study involving subjects who received beremagene geperpavec via skin swabs, no viral vector DNA was detected in blood or urine.4
- Half-life
There is no information available.
- Clearance
There is no information available.
- Adverse Effects
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There is no information available regarding acute toxicity and overdosage of beremagene geperpavec.
- Pathways
- Not Available
- Pharmacogenomic Effects/ADRs
- Not Available
Interactions
- Drug Interactions
- This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.Not Available
- Food Interactions
- No interactions found.
Products
Drug product information from 10+ global regionsOur datasets provide approved product information including:dosage, form, labeller, route of administration, and marketing period.Access drug product information from over 10 global regions.- Brand Name Prescription Products
Name Dosage Strength Route Labeller Marketing Start Marketing End Region Image Vyjuvek Kit; Suspension 5000000000 [PFU]/1mL Topical Krystal Biotech, Inc. 2023-05-19 Not applicable US 
Categories
- Drug Categories
- Classification
- Not classified
- Affected organisms
- Not Available
Chemical Identifiers
- UNII
- AQN7K24KQU
- CAS number
- 2241888-62-8
References
- General References
- 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: In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial. Nat Med. 2022 Apr;28(4):780-788. doi: 10.1038/s41591-022-01737-y. Epub 2022 Mar 28. [Article]
- Shinkuma S: Dystrophic epidermolysis bullosa: a review. Clin Cosmet Investig Dermatol. 2015 May 26;8:275-84. doi: 10.2147/CCID.S54681. eCollection 2015. [Article]
- Guide SV, Gonzalez ME, Bagci 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: Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa. N Engl J Med. 2022 Dec 15;387(24):2211-2219. doi: 10.1056/NEJMoa2206663. [Article]
- FDA Approved Drug Products: VYJUVEK (beremagene geperpavec-svdt) biological suspension mixed with excipient gel for topical application [Link]
- BioSpace: Krystal Biotech Receives FDA Approval for the First-Ever Redosable Gene Therapy, VYJUVEK™ (beremagene geperpavec-svdt) for the Treatment of Dystrophic Epidermolysis Bullosa [Link]
- External Links
- 2637758
- Wikipedia
- Beremagene_geperpavec
Clinical Trials
- Clinical Trials
Phase Status Purpose Conditions Count 3 Completed Treatment DEB - Dystrophic Epidermolysis Bullosa / Dominant Dystrophic Epidermolysis Bullosa / Dystrophic Epidermolysis Bullosa / Recessive Dystrophic Epidermolysis Bullosa 1 3 Completed Treatment Dominant Dystrophic Epidermolysis Bullosa / Dystrophic Epidermolysis Bullosa / Recessive Dystrophic Epidermolysis Bullosa 1 1, 2 Completed Treatment Dystrophic Epidermolysis Bullosa 1
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Not Available
- Dosage Forms
Form Route Strength Kit; suspension Topical 5000000000 [PFU]/1mL - Prices
- Not Available
- Patents
- Not Available
Properties
- State
- Not Available
- Experimental Properties
- Not Available
Drug created at May 26, 2023 19:11 / Updated at June 23, 2023 10:36