Drug intelligence / Profile preview

pegloticase + azathioprine

Development stage
Unknown
Lead developer
Amgen
Modality
Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Small Molecules
Administration
Intravenous (pegloticase), Oral (azathioprine)
01

Overview

Pegloticase + azathioprine is a **combination therapy** used for the management of **refractory or uncontrolled gout**. Pegloticase is a PEGylated recombinant uricase enzyme that catalyzes the oxidation of uric acid to allantoin, a highly water-soluble compound that is readily excreted by the kidneys, thus lowering serum urate levels. However, pegloticase monotherapy is associated with high immunogenicity, frequently leading to the formation of anti-drug antibodies and loss of efficacy as well as increased risk of infusion reactions. Azathioprine, an immunosuppressant purine analog, is co-administered to reduce the immunogenic response by suppressing antibody formation against pegloticase, thereby improving response rates, durability, and safety. This combination has been specifically studied in patients with uncontrolled gout who failed or were intolerant to other urate-lowering therapies, showing increased efficacy (persistent urate lowering and reduced infusion reactions) compared to pegloticase monotherapy[1][3][7].

02

Targets

UOX (Urate oxidase)

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