CUTX-101 (Copper Histidinate)
CUTX-101 (Copper Histidinate) is in development to restore copper homeostasis and maintain copper levels in patients with Menkes disease and related disorders. CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured under current Good Manufacturing Practice (cGMP) that is intended to improve tolerability due to its physiological pH, and to bypass the oral absorption of copper, which is impaired in patients with Menkes disease. In the Phase 1/2 clinical study conducted at NICHD, early treatment of Menkes patients with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival.
In October 2021, Cyprium announced positive results from a safety and efficacy analysis of data integrated from two completed pivotal studies in patients with Menkes disease treated with CUTX-101. These data were presented as a virtual poster at the 2021 American Academy of Pediatrics National Conference & Exhibition.
In December 2021, Cyprium initiated a rolling submission of a New Drug Application to the U.S. Food and Drug Administration (FDA) for CUTX-101 for the treatment of Menkes disease.
CUTX-101 has been granted orphan drug designation by the FDA. The FDA granted Breakthrough Therapy Designation, Fast Track Designation and Rare Pediatric Disease Designation to CUTX-101. In July 2020, European Medicines Agency (EMA) Committee for Orphan Medicinal Products issued a positive opinion on the Orphan Medicinal Product Designation for CUTX-101.
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