Sentynl Therapeutics Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved ZYCUBO® (copper histidinate) for the treatment of patients with Menkes disease, a rare, inherited disorder that affects copper absorption and distribution in the body.
ZYCUBO® is indicated for subcutaneous administration and is designed to address the underlying copper deficiency associated with Menkes disease, a condition that can lead to severe neurological impairment and is often fatal if left untreated. The approval represents an important milestone for patients and families affected by this ultra-rare disorder, for which treatment options have historically been limited.
“With the FDA approval of ZYCUBO®, we are delivering a regulated, FDA-approved therapy to a community that has long relied on unapproved or compounded treatment options,” said a spokesperson for Sentynl Therapeutics. “This approval underscores our commitment to advancing therapies for patients with rare and underserved diseases.”
Menkes disease is caused by mutations in the ATP7A gene, resulting in impaired copper transport. Early diagnosis and timely initiation of copper replacement therapy are critical to improving clinical outcomes. ZYCUBO® provides a standardized, pharmaceutical-grade formulation of copper histidinate, offering clinicians and caregivers a consistent and approved treatment option.
Sentynl Therapeutics focuses on acquiring, developing, and commercializing treatments for rare diseases, with an emphasis on addressing unmet medical needs. The approval of ZYCUBO® further strengthens the company’s rare disease portfolio and highlights its role in bringing niche therapies through the regulatory pathway to patients in need.
ZYCUBO® is expected to be made available to eligible patients in the U.S. through established specialty distribution channels, supported by patient access and assistance programs to help families navigate treatment initiation and continuity of care.
The FDA approval marks a significant advancement in the management of Menkes disease and reflects continued progress in rare disease drug development aimed at improving survival and quality of life for affected patients.
