Summary

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• This summary is based on the review of one randomized controlled trial. ^[1]
• The study demonstrated that elapegademase-lvlr effectively maintained sufficient adenosine deaminase (ADA) activity, with all four patients achieving undetectable levels of deoxyadenosine nucleotides (dAXPs) and increased T and B cell numbers, alongside slightly elevated Immunoglobulin M (IgM) and Immunoglobulin A (IgA) immunoglobulin levels.
• Elapegademase-lvlr showed comparable efficacy to pegademase, the previous enzyme-replacement therapy for ADA deficiency, as indicated by the monitoring of trough dAXP levels (≤0.02 μmol/mL) and serum ADA activity (≥1100 U/L).
• The study included patients aged 0-25 years, with one infant patient receiving elapegademase at 0.4 mg/kg/week until death from pneumonia caused by cytomegalovirus infection, while three other patients received a maximum dose of 0.3 mg/kg/week for 164-169 weeks, completing the study with effective biochemical control.
• One infant patient died from pneumonia caused by cytomegalovirus infection during the study, but this adverse event was assessed as unrelated to elapegademase-lvlr; serious adverse events occurred in three other patients, all deemed unrelated to the treatment.
• No drug-related adverse events were reported, and the safety profile of elapegademase-lvlr was found to be comparable to that of pegademase.
• There is no population types or subgroups information available in the reviewed studies.