Copper histidinate is a copper-histidine complex indicated as a replacement therapy for the treatment of Menkes disease to increase serum copper and ceruloplasmin levels. Menkes disease is a rare, X-linked recessive disorder caused by pathogenic variants in the ATP7A gene, which encodes a copper transport protein essential for absorbing dietary copper and transporting it across the blood-brain barrier.
- Mechanism
- Curator reviewed · 3 references
Copper histidinate is a copper replacement therapy that is administered via subcutaneous injection to bypass the impaired gastrointestinal absorption characteristic of patients with Menkes disease. Once in the circulation, the copper-histidine complex can utilize L-type amino acid transporter 1 (LAT1 or SLC7A5) - a transporter responsible for supplying essential amino acids to the brain and other tissues - to enter cells. This mechanism allows for effective copper delivery to tissues despite the absence of functional ATP7A transporters.
- Primary indication
- Copper histidinate is indicated for the treatment of Menkes disease in pediatric patients.Curator reviewed · 1 structured indication
- Formula / weight
- C12H16CuN6O4 · 371.844 g/mol (avg)
- First approval
- United States, 2026
- Also known as
- Copper bis(histidinate) · Copper histidine
- Brand names
Resolves to
UUYDYUZBCIHUFZ-MDTVQASCSA-LSMILES[Cu++].[H][C@](N)(CC1=CN=CN1)C([O-])=O.[H][C@](N)(CC1=CN=CN1)C([O-])=OWhat you can answer from here — as of September 01, 2026
Which drugs share a target with Copper histidinate, and which of those have an active Phase 3 trial?
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