fidanacogene elaparvovec-dzkt

Action and Spectrum

Actions and Spectrum:  

Actions:  

Fidanacogene elaparvovec-dzkt is a gene therapy treatment that helps in improving the levels of clotting factor IX (FIX) in the disease hemophilia B. The drug delivers a copy of the FIX gene, which is responsible for fixing blood clotting factor directly into the cells of the patient. 

Drug Interaction

Adverse Reaction

Adverse reaction 

Frequency defined 

>10% 

Elevated transaminases (13.3-53.3%) 

Black Box Warning

Black Box Warning  

None

Contraindication / Caution

Contraindication/Caution:  

Contraindications 

None  

Cautions 

Hepatotoxicity 

Malignancy 

Infusion reactions 

Pregnancy / Lactation

Pregnancy & breastfeeding: 

Pregnancy consideration:   

No data is available regarding the administration of the drug during pregnancy.  

Breastfeeding warnings:   

No data is available regarding the excretion of drugs in breast milk.  

Pregnancy category:  

Category A: well-controlled and satisfactory studies show no risk to the fetus in the first or later trimester.  

Category B: There was no evidence of risk to the fetus in animal studies, and there were not enough studies on pregnant women.  

 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: No data for the drug under this category is available.  

Pharmacology

Pharmacology: The drug works as a gene therapy. 

Pharmacodynamics:  

The mechanism of action of fidanacogene elaparvovec-dzkt is the insertion of a functional copy of the factor IX (FIX) gene into the patient cells.  

For hemophilia, the lack of the clotting factor FIX, which helps fixing the blood, is the determinant feature. The major goal of this therapy is to correct patients by providing a functional copy of a gene that produces FIX, thus contributing to the normal clotting. 

Pharmacokinetics:  

Distribution: 

The infused viral vector containing factor IX gene, after the delivery of transfected factor IX gene, enters the target cells to establish the integration of the new gene. The ways the viral vector is distributed within the body vary by, among others like tissue tropism and circulation. 

Adminstartion

Administration:  

The drug is administered intravenously. 

Patient Information Leaflet

Patient information leaflet  

Generic Name: fidanacogene elaparvovec-dzkt 

Why do we use fidanacogene elaparvovec-dzkt?  

Fidanacogene elaparvovec-dzkt, is a gene therapy intended for use in treating adults with moderate to severe hemophilia B (congenital Factor IX deficiency).