name: | Pegademase |
ATC code: | L03AX04 | route: | intramuscular |
n-compartments | 1 |
Pegademase (PEG-ADA) is a modified form of adenosine deaminase enzyme conjugated with polyethylene glycol to increase its stability and half-life. It has been used as enzyme replacement therapy for severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency. It is an FDA-approved orphan drug but less commonly used since the advent of stem cell and gene therapies.
Estimated pharmacokinetic parameters in ADA-SCID adult patients based on typical literature-reported dosing and known characteristics of PEGylated proteins. No published human pharmacokinetic data found for pegademase; values are approximated/extrapolated from available enzyme replacement therapy data.