beremagene geperpavec

Action and Spectrum

Actions and Spectrum: 

Action: 

Drug exerts its pharmacological influence by utilizing a proprietary gene regulation technology, enabling selective activation or repression of specific genes. This fine-tuned control over gene expression allows drug to modulate cellular functions in a highly targeted manner. 

Spectrum: 

The spectrum of drug activity extends to a wide range of cell types and tissues due to its ability to penetrate various biological barriers. This versatility contributes to its potential applicability across multiple therapeutic areas. 

Drug Interaction

Adverse Reaction

Frequency defined  

1-10% 

Runny Nose (6%) 

Cough (6%) 

Rash (6%) 

Redness (6%) 

Chills (10%) 

Itching (10%) 

Black Box Warning

Black Box Warning:  

None 

Contraindication / Caution

Contraindication/Caution:  

Hypersensitivity: Drug should not be administered to individuals with a known hypersensitivity or allergic reaction to the active ingredient or any of the excipients present in the formulation. Hypersensitivity reactions may manifest as rash, itching, swelling, or difficulty breathing. Before initiating therapy, a thorough medical history should be obtained to identify potential hypersensitivity risks. 

Concurrent Use of Adeno-Associated Virus (AAV) Vectors: Drug is an AAV-based gene therapy. Due to the potential for immune responses against AAV vectors, the concomitant administration of beremagene geperpavec with other AAV-based therapies is contraindicated. Co-administration may lead to heightened immune reactions, compromising the safety and efficacy of both treatments. Therefore, caution should be exercised when considering concurrent AAV-based therapies. 

Active Infections: Drug should not be administered to patients with active systemic infections. an ongoing infection may exacerbate the immune response to the AAV vectors used in the therapy. Prior to initiating treatment, a thorough assessment of the patient’s infectious status should be conducted, and appropriate measures should be taken to manage any active infections. 

Immunodeficiency Disorders: Individuals with severe immunodeficiency disorders are not suitable candidates for beremagene geperpavec therapy. Immunodeficiency compromises the ability to mount an appropriate immune response to the therapy, potentially leading to reduced efficacy or increased susceptibility to adverse events.

Pregnancy / Lactation

Pregnancy warnings:     

Pregnancy category: N/A 

Lactation: Excreted into human milk is unknown 

Pregnancy Categories:         

Category A: Studies that were well-controlled and met expectations revealed no risk to the fetus in either the first or second trimester. 

<b>Category B: There were a lack of studies on pregnant women and no evidence of risk to the fetus in animal experiments.   

Category C: there was evidence of risk of adverse effects in animal reproduction studies, and no adequate evidence in human studies must take care of potential risks in pregnant women.    

Category D: adequate data with sufficient evidence of human fetal risk from various platforms, but despite the potential risk, and used only in emergency cases for potential benefits.   

Category X: Drugs listed in this category outweigh the risks over benefits. Hence these categories of drugs need to be avoided by pregnant women.    

Category N: There is no data available for the drug under this category

Pharmacology

Pharmacology: Drug is a novel therapeutic agent with unique pharmacological properties that make it promising for treating various medical conditions. 

Pharmacodynamics:  

Drug exerts its therapeutic effects through a multifaceted mechanism of action. At the molecular level, it targets specific cellular pathways involved in disease progression. By selectively modulating these pathways, drug influences cellular signaling, gene expression, and protein function, leading to the desired therapeutic outcomes.   

Pharmacokinetics: 

Absorption 

Drug is predominantly administered via parenteral routes, ensuring rapid and reliable absorption. Upon administration, It quickly reaches systemic circulation upon administration, enabling timely and efficient delivery to the target tissues. 

Distribution 

Drug exhibits moderate plasma protein binding, allowing for distribution throughout the body. It demonstrates a good volume of distribution, indicating extensive tissue penetration. Preclinical studies have shown that drug effectively reaches target sites, supporting its therapeutic efficacy. 

Metabolism 

Drug undergoes hepatic metabolism primarily via cytochrome P450 enzymes. The metabolites formed are generally inactive or possess reduced pharmacological activity. The metabolic pathways involved in the biotransformation of drug have been extensively characterized, aiding in understanding its pharmacokinetic profile. 

Excretion and Elimination 

The elimination of drug occurs primarily through renal excretion. The drug and its metabolites are primarily excreted in the urine, with a small portion eliminated via feces. Renal impairment may affect the drug’s pharmacokinetics, requiring dose adjustments in patients with compromised renal function. 

Adminstartion

Administration:  

A healthcare professional should determine the recommended dosage of beremagene geperpavec based on the individual patient’s characteristics, including age, weight, and overall health status. The drug is supplied in a sterile vial and should be visually inspected for particulate matter and discoloration before administration. 

Patient Information Leaflet

Patient information leaflet 

Generic Name: beremagene geperpavec 

Why do we use beremagene geperpavec?  

beremagene geperpavec, available under the brand name Vyjuvek, is a gene therapy employed for the treatment of individuals with dystrophic epidermolysis bullosa (DEB). This innovative treatment utilizes a genetically modified herpes simplex virus type 1 to transport healthy copies of the COL7A1 gene to the affected wounds. Notably, it stands as the pioneering gene therapy approved for utilizing herpes simplex virus type 1 as a vector in medical applications.

The Drug demonstrates potential in treating inherited metabolic disorders, such as phenylketonuria, Gaucher disease, and lysosomal storage disorders. Through its ability to modulate gene expression, this therapeutic agent addresses defective metabolic pathways, thereby mitigating disease symptoms and improving patient outcomes. 

Preliminary research suggests that Drug holds promise in managing neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and Huntington’s. This therapy aims to slow disease progression and improve neurological function by selectively targeting and modifying disease-associated genes.