Menkes Disease Treatments
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Brand Name
Zycubo
Generic Name
Histidinate
View Brand Information FDA approval date: January 12, 2026
Form: Injection
What is Zycubo (Histidinate)?
ZYCUBO is indicated for the treatment of Menkes disease in pediatric patients. ZYCUBO is a copper replacement product indicated for the treatment of Menkes disease in pediatric patients. Limitations of Use ZYCUBO is not indicated for the treatment of Occipital Horn Syndrome. Limitations of Use ZYCUBO is not indicated for the treatment of Occipital Horn Syndrome.
Approved To Treat
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Related Clinical Trials
Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease: Double-blind Placebo-controlled Randomized Crossover Clinical Trial.
Summary: This clinical trial will evaluate the safety, tolerability, dosing, and efficacy of Northera (Droxidopa) in children with Menkes disease aged 7 to 17 years who survived the major neurodegenerative and neurocognitive effects of Menkes disease through early Copper Histidinate treatment. The investigator hypothesizes that Northera (Droxidopa) treatment in pediatric Menkes disease survivors with sympt...
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Brand Information
Zycubo (copper histidinate)
1INDICATIONS AND USAGE
ZYCUBO is indicated for the treatment of Menkes disease in pediatric patients.
2DOSAGE FORMS AND STRENGTHS
For Injection: 2.9 mg of copper histidinate (equivalent to 0.5 mg elemental copper) as a blue lyophilized powder or cake in a single-dose vial for reconstitution.
3CONTRAINDICATIONS
None.
4ADVERSE REACTIONS
The following clinically significant adverse reactions are described elsewhere in the labeling:
- Copper Accumulation and Risk of Toxicity: Drug-Induced Kidney Injury, Liver Dysfunction, Hematological Abnormalities
4.1Clinical Trials Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
The pooled safety analysis from 2 open-label, single-arm clinical trials included a total of 129 ZYCUBO-treated patients with an age range from 0 to 48 months. Patients less than 1 year of age received ZYCUBO 1.45 mg twice daily, and patients 1 year of age and older received ZYCUBO 1.45 mg once daily. The median exposure duration was 24 months (range: 1 to 39 months)
5DESCRIPTION
ZYCUBO (copper histidinate) for injection is a copper replacement product. The chemical name is copper, (L-histidinato-ϰ
The chemical structure is:
ZYCUBO is a sterile, preservative-free, blue lyophilized powder or cake for subcutaneous injection after reconstitution with 1 mL sterile 0.9% Sodium Chloride Injection, USP. Each single-dose vial contains 2.9 mg of copper histidinate (equivalent to 0.5 mg elemental copper). The resultant solution has a concentration of 2.9 mg/mL and a pH of 7.4.
6CLINICAL STUDIES
The efficacy of ZYCUBO was evaluated in pediatric patients with Menkes disease (age at treatment initiation ranges 0.1 to 31.4 months) receiving 3 years of copper histidinate treatment in two open-label, single-arm clinical trials (Trial 1, NCT00001262 and Trial 2, NCT00811785). Data from ZYCUBO-treated patients in these two trials were compared to data from an untreated contemporaneous external control cohort as collected under a protocol amendment of Trial 2. In both trials, pediatric patients:
- Less than 1 year of age received 1.45 mg of ZYCUBO administered subcutaneously twice daily until 1 year of age.
- Equal to or greater than 1 year of age received 1.45 mg of ZYCUBO subcutaneously once daily for up to 3 years.
Overall survival was evaluated in a subset of the pooled population from Trial 1 and Trial 2, referred to as the pooled efficacy population. This efficacy population included only patients with Menkes disease who carried a severe pathogenic variant of the
Patients in the pooled efficacy population were assigned to 1 of 4 cohorts as described in
In the 4 cohorts, 81 patients were male (98%) except for 2 female (2%) patients in ZYCUBO-LT. The pooled efficacy population included patients with the following race and ethnicity: 52 White (63%), 11 Hispanic (13%), 8 Black or African American (10%), 6 Unknown (7%), 4 Other (5%), and 2 Asian or Pacific Islander (2%). The majority of patients in all 4 cohorts were born prematurely: ZYCUBO-ET = 77%, ZYCUBO-LT = 66%, EC-ET = 82%, and EC-LT = 81%.
7PATIENT COUNSELING INFORMATION
Advise the patient and/or caregiver to read the FDA-approved patient labeling (