Elapegademase-lvlr
Elapegademase-lvlr Uses, Dosage, Side Effects, Food Interaction and all others data.
Elapegademase-lvlr is a PEGylated recombinant adenosine deaminase. It can be defined molecularly as a genetically modified bovine adenosine deaminase with a modification in cysteine 74 for serine and with about 13 methoxy polyethylene glycol chains bound via carbonyl group in alanine and lysine residues. Elapegademase-lvlr is generated in E. coli, developed by Leadiant Biosciences and FDA approved on October 5, 2018.
In clinical trials, elapegademase was shown to increase adenosine deaminase activity while reducing the concentrations of toxic metabolites which are the hallmark of ADA-SCID. As well, it was shown to improve the total lymphocyte count.
| Trade Name | Elapegademase-lvlr |
| Availability | Prescription only |
| Generic | Elapegademase |
| Elapegademase Other Names | Elapegademase, Elapegademase-lvlr |
| Related Drugs | Revcovi |
| Type | Intramuscular |
| Formula | C1797H2795N477O544S12 |
| Weight | 115000.0 Da |
| Protein binding | This pharmacokinetic property is not significant as the main effect is in the blood cells. |
| Groups | Approved |
| Therapeutic Class | |
| Manufacturer | |
| Available Country | United States |
| Last Updated: | September 19, 2023 at 7:00 am |
Uses
Elapegademase-lvlr is a recombinant adenosine deaminase used to treat adenosine deaminase severe combined immune deficiency.
Elapegademase-lvlr is approved for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients. This condition was previously treated by the use of bovine pegamedase as part of an enzyme replacement therapy.
ADA-SCID is a genetically inherited disorder that is very rare and characterized by a deficiency in the adenosine deaminase enzyme. The patients suffering from this disease often present with a compromised immune system. This condition is characterized by very low levels of white blood cells and immunoglobulin levels which results in severe and recurring infections.
Elapegademase-lvlr is also used to associated treatment for these conditions: Severe Combined Immunodeficiency Syndrome caused by Adenosine Deaminase Deficiency
How Elapegademase-lvlr works
The ADA-SCID is caused by the presence of mutations in the ADA gene which is responsible for the synthesis of adenosine deaminase. This enzyme is found throughout the body but it is mainly active in lymphocytes. The normal function of adenosine deaminase is to eliminate deoxyadenosine, created when DNA is degraded, by converting it into deoxyinosine. This degradation process is very important as deoxyadenosine is cytotoxic, especially for lymphocytes. Immature lymphocytes are particularly vulnerable as deoxyadenosine kills them before maturation making them unable to produce their immune function.
Therefore, based on the causes of ADA-SCID, elapegademase works by supplementing the levels of adenosine deaminase. Being a recombinant and an E. coli-produced molecule, the use of this drug eliminates the need to source the enzyme from animals, as it was previously.
Toxicity
As elapegademase is a therapeutic protein, there is a potential risk of immunogenicity.
There are no studies related to overdose but the highest weekly prescribed dose in clinical trials was 0.4 mg/kg. In nonclinical studies, a dosage of 1.8 fold of the clinical dose produced a slight increase in the activated partial thromboplastin time.
Elapegademase-lvlr Disease Interaction
Volume of Distribution
This pharmacokinetic property has not been fully studied.
Elimination Route
Elapegademase-lvlr is administered intramuscularly and the reported Tmax, Cmax and AUC are approximately 60 hours, 240 mmol.h/L and 33000 hr.mmol/L as reported during a week.
Half Life
This pharmacokinetic property has not been fully studied.
Clearance
This pharmacokinetic property has not been fully studied.
Elimination Route
This pharmacokinetic property has not been fully studied.
Innovators Monograph
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