![]() Three patients had transient, non-neutralizing antibodies to pegademase, elapegademase, and/or polyethylene glycol ≤ 47 weeks of treatment without effect on trough plasma ADA activity or trough erythrocyte deoxyadenosine nucleotide levels. Elapegademase had a comparable safety profile to pegademase no patient developed a severe infectious complication. Total lymphocyte counts increased for all completer patients from between 1.2- and 2.1-fold at the end of study compared with baseline. ![]() In these patients, elapegademase improved ADA activity compared with pegademase and maintained metabolic detoxification. The 6 remaining patients completed 71−216 weeks of elapegademase therapy with a formulation that did not contain EDTA. One patient withdrew after 2 doses of an early elapegademase formulation due to injection-site pain caused by EDTA. Maintenance of metabolic detoxification and adequate ADA activity were evaluated periodically. MethodsĪfter once-weekly pegademase dosage was adjusted to achieve therapeutic metabolic detoxification and trough ADA activity, patients transitioned to a bioequivalent dose of elapegademase. This study was a 1-way crossover from pegademase to elapegademase in 7 patients with ADA-SCID to assess efficacy and safety outcomes for elapegademase. ![]() Elapegademase is a PEGylated recombinant bovine ADA ERT developed to replace the now-discontinued bovine-derived pegademase. Metabolic detoxification with enzyme replacement therapy (ERT) promotes immune recovery in patients with adenosine deaminase (ADA)–deficient severe combined immunodeficiency (ADA-SCID). ![]()
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