CUTX-101 FDA Approval Status
Last updated by Judith Stewart, BPharm on May 27, 2025.
FDA Approved: No
Brand name: CUTX-101
Generic name: copper histidinate
Dosage form: Injection
Company: Sentynl Therapeutics, Inc.
Treatment for: Menkes Disease
CUTX-101 (copper histidinate) is a copper replacement therapy in development for the treatment of Menkes disease.
- Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A. The condition is characterized by distinctive clinical features, including sparse and depigmented hair (“kinky hair”), connective tissue problems, and severe neurological symptoms such as seizures, hypotonia, failure to thrive, and neurodevelopmental delays.
- CUTX-101 is a subcutaneous injectable formulation of copper histidinate that is intended to improve tolerability due to its physiological pH. Copper histidine works in the treatment of Menkes disease by delivering bioavailable copper directly into the bloodstream. The ATP7A mutations in Menkes disease impact the transport of copper to a range of organs and systems, such as the lungs, brain and heart.
- The New Drug Application (NDA) for CUTX-101 was supported by positive topline clinical efficacy results that demonstrated statistically significant improvement in overall survival for Menkes disease patients who received early treatment with CUTX-101, with a nearly 80% reduction in the risk of death compared to an untreated historical control cohort. Median overall survival was 177.1 months for CUTX-101 early treatment cohort compared to 16.1 months for the untreated historical control cohort.
- CUTX-101 has been granted Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug Designations by the FDA. The Prescription Drug User Fee Act (PDUFA) target action date is June 30, 2025.
Development timeline for CUTX-101
Further information
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