Drug class

Urea Cycle Disorder Agents

Urea cycle disorder agents are a therapeutic class of metabolic medications primarily functioning as nitrogen-scavenging compounds or synthetic enzyme activators. Read more about Urea Cycle Disorder Agents

25 drugs in this class 22 manufacturers

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Drugs in Urea Cycle Disorder Agents

Showing 1–9 of 25 drugs

CARBAGLU (carglumic acid)

UREA CYCLE DISORDER AGENTS · HYPERAMMONEMIA ASSOCIATED WITH N-ACETYLGLUTAMATE SYNTHASE DEFICIENCY ·+2
ORPHAN EUROPE/R

SODIUM PHENYLBUTYRATE

UREA CYCLE DISORDER AGENTS · CARBAMOYL PHOSPHATE SYNTHETASE DEFICIENCY ·+2
PAR HEALTH USA,

SODIUM PHENYLBUTYRATE

UREA CYCLE DISORDER AGENTS · CARBAMOYL PHOSPHATE SYNTHETASE DEFICIENCY ·+2
PAR HEALTH USA,
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About Urea Cycle Disorder Agents.

Urea cycle disorder agents are a therapeutic class of metabolic medications primarily functioning as nitrogen-scavenging compounds or synthetic enzyme activators.

These agents, which include sodium phenylbutyrate, glycerol phenylbutyrate, and carglumic acid, work by providing alternative metabolic pathways for waste nitrogen excretion or by restoring critical enzymatic function to prevent the accumulation of neurotoxic ammonia.

They are clinically indicated for the acute management and long-term maintenance of inherited urea cycle disorders—such as ornithine transcarbamylase, carbamoyl phosphate synthetase, and argininosuccinate synthetase deficiencies—typically serving as adjunctive therapy alongside dietary protein restriction to prevent hyperammonemic crises and permanent neurological damage.