Elapegademase is a recombinant adenosine deaminase used to treat adenosine deaminase severe combined immune deficiency. 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...
- Mechanism
- Curator reviewed · 2 references
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.
- Primary indication
- Elapegademase is approved for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.Curator reviewed · 1 structured indication
- Formula / weight
- C1797H2795N477O544S12 · 115000.0 Da
- First approval
- United States, 2018
- Also known as
- (74-SERINE (C>S),245-THREONINE (A>T))BOS TAURUS ADENOSINE DEAMINASE (BOVINE ADENOSINE AMINOHYDROLASE, EC=3.5.4.4)-(1-356)-PEPTIDE PRODUCED IN ESCHERICHIA COLI, AN AVERAGE OF 13 RESIDUES ARE SUBSTITUTED ON N.ALPHA..1ALANINE AND N6 OF LYSINE RESIDUES WITH · (CYS74>SER,ALA245>THR)ADENOSINE DEAMINASE (BOS TAURUS, BOVINE)-(1-356)-PEPTIDE, PRODUCED IN ESCHERICHIA COLI, SUBSTITUTED ON N2 OF THE N-TERMINAL ALANYL RESIDUE (A1) AND ON N6 OF LYSYL RESIDUES (K) WITH AN AVERAGE OFAPPROXIMATELY 13 .OMEGA.-METHOXYPOLY(O · Elapegademase-lvlr · monomethoxypolyethylene glycol recombinant adenosine deaminase · POLY(OXY-1,2-ETHANEDIYL), .ALPHA.-CARBOXY-.OMEGA.-METHOXY-, AMIDE WITH ADENOSINE DEAMINASE (SYNTHETIC)
- Code names
- EZN-2279
- Brand names
Resolves to
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