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Elapegademase is a PEGylated recombinant adenosine deaminase enzyme replacement therapy used to treat adenosine deaminase severe combined immune deficiency (ADA-SCID), a rare inherited disorder that results in profound immunodeficiency. The drug is produced in E. coli and consists of genetically modified bovine adenosine deaminase with specific amino acid modifications and multiple methoxy polyethylene glycol chains attached. Elapegademase works by supplementing deficient ADA activity, thereby reducing toxic levels of adenosine and deoxyadenosine nucleotides, which are particularly harmful to lymphocytes. This leads to improved immune function and increased lymphocyte numbers in patients with ADA-SCID[1][2][3][4][5][6][8]. It was developed by Leadiant Biosciences and received FDA approval in 2018[2].
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