elapegademase

Action and Spectrum

Actions and Spectrum: 

The spectrum of elapegademase treats Adenosine Deaminase-Severe Combined Immune Deficiency (ADA-SCID) in adults and children. It is a rare disorder where the body cannot produce a specific enzyme that is essential for the proper function of the immune system. 

The action of elapegademase is to substitute or supplement the missing enzyme and help improve the function of the immune system, which can help to reduce the risk of infections and improve overall health outcomes. 

Drug Interaction

Adverse Reaction

>10% 

Cough (50%) 

Vomiting (33%) 

Frequency undefined: 

Arthralgia 

Conjunctivitis 

Diarrhea 

Fatigue 

Galt disturbance 

Groin abscess 

Hematochezia 

Laceration 

Nausea 

Oral candidiasis 

Upper respiratory tract infection 

Black Box Warning

Black Box Warning: 

It is essential to follow the dosage and administration instructions for elapegademase carefully, as an overdose can cause serious side effects. 

Contraindication / Caution

Contraindication/Caution: 

elapegademase should not be used in people hypersensitive to the drug itself. 

Pregnancy / Lactation

Pregnancy consideration:  

Insufficient data regarding the drug’s effect on the fetus during pregnancy is available.  

Breastfeeding warnings:  

The distribution of the drug in breast milk is unknown.  

Pregnancy category: 

• Category A: well-controlled and Satisfactory studies do not show risk to the fetus in the first/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 available 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 is available for the drug under this category. 

Pharmacology

Pharmacology 

elapegademase is a recombinant human adenosine deaminase (ADA) enzyme replacement therapy. It is designed to substitute for or supplement the missing ADA enzyme in individuals with ADA-SCID. This rare genetic disorder affects the immune system and leads to severe, recurrent infections. 

Pharmacodynamics: 

When elapegademase is administered intramuscularly, it is rapidly distributed throughout the body and binds to the cells that lack ADA. Once bound, the enzyme converts the toxic substrate adenosine to inosine. It helps to allow normal metabolism to proceed. 

By supplementing the missing enzyme, elapegademase helps prevent the accumulation of toxic adenosine and deoxyadenosine. Hence, it helps reduce the risk of infections and improve overall health outcomes. Also, elapegademase can also improve T-cell counts, which may reduce the risk of fatal infections and malignancy. 

Pharmacokinetics: 

The half-life of elapegademase in the body is relatively long due to the addition of polyethylene glycol (PEG) to the enzyme, which slows its clearance from the body. The half-life of elapegademase is about 12-16 hours, which allows for once-weekly dosing. 

Absorption 

elapegademase is administered by injection and is absorbed into the bloodstream. 

Distribution 

After it is absorbed, elapegademase is distributed throughout the body and can be found in high liver, spleen, and bone marrow concentrations. 

Metabolism 

The metabolism of elapegademase occurs primarily in the liver. There it is metabolized into several active and inactive metabolites.  

Elimination and excretion 

These metabolites are then eliminated from the body through the kidneys and feces. 

Adminstartion

Administration: 

The elapegademase is administered through the intramuscular route

Patient Information Leaflet

Patient information leaflet 

Generic Name: elapegademase 

Pronounced: EL-a-peg-AD-e-mase 

Why do we use elapegademase? 

elapegademase treats ADA deficiency