Mumbai, Jan. 13 -- This marks the first and only FDA-approved therapy for this rare and fatal genetic disorder in the United States. ZYCUBO(R) is not indicated for Occipital Horn Syndrome.

Menkes disease, an X-linked recessive pediatric condition caused by mutations in the ATP7A gene, prevents patients from absorbing dietary copper, resulting in impaired copper transport across the blood-brain barrier. Untreated patients typically do not survive beyond three years of age.

ZYCUBO is a subcutaneous injectable formulation that restores copper homeostasis and maintains copper levels in patients. Clinical trials demonstrated a statistically significant improvement in overall survival, with the early treatment cohort showing median survival o...