Sentynl Therapeutics, a Zydus Lifesciences subsidiary, announced the FDA’s acceptance of its New Drug Application (NDA) resubmission for copper histidinate (CUTX-101) to treat pediatric patients with Menkes disease. The FDA granted a Class I response, setting a new PDUFA date of January 14, 2026. If approved, CUTX-101 will be the first FDA-approved treatment for this rare genetic disorder, which affects approximately 1 in 34,810 to 1 in 8,664 live male births.
CUTX-101 Receives FDA Nod
Sentynl Therapeutics, Inc., wholly-owned by Zydus Lifesciences Ltd., has received acceptance from the U.S. Food and Drug Administration (FDA) for its resubmitted New Drug Application (NDA) for copper histidinate (CUTX-101). This drug is intended for the treatment of Menkes disease in pediatric patients.
Revised PDUFA Date
The FDA has designated the resubmission as a Class I response, setting a new Prescription Drug User Fee Act (PDUFA) target action date of January 14, 2026.
Menkes Disease Treatment
CUTX-101, if approved, would become the first and only FDA-approved treatment for Menkes disease, a rare X-linked recessive pediatric genetic disorder. This condition affects an estimated 1 in 34,810 to as high as 1 in 8,664 live male births. It is caused by mutations of the copper transporter ATP7A.
About CUTX-101
CUTX-101 is an investigational drug that has been formulated as a subcutaneous injectable using cGMP standards, intended to improve tolerability through its physiological pH. A Phase 1/2 clinical trial showed that early treatment with CUTX-101 led to improvements in both neurodevelopmental outcomes and overall survival.
Clinical Trial Results
Clinical trials for CUTX-101 have demonstrated statistically significant improvements in overall survival for Menkes disease subjects who received early treatment compared to an untreated historical control cohort, showcasing a nearly 80% reduction in the risk of death. The median overall survival was 177.1 months for the early treatment group, compared to 16.1 months for the untreated historical control group.
Source: BSE