Fortress Biotech receives FDA approval for Zycubo to treat Menkes disease

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Source: Pharmaceutical Technology

Original: https://www.pharmaceutical-technology.com/news/fortress-biotech-fda-approval-zyc...

Published: Wed, 14 Jan 2026 09:28:33 +0000

The FDA has approved Zycubo (copper histidinate) as the first treatment for Menkes disease in children[1][2]. Menkes disease is a rare genetic disorder caused by mutations in the ATP7A gene that leads to impaired absorption and transport of copper in the body[3]. Zycubo is given as an injection under the skin and delivers copper in a form that bypasses a genetic defect in intestinal absorption[4]. In clinical trials with early initiation of Zycubo treatment, a median overall survival of 177.1 months was achieved compared to 17.6 months in untreated patients, representing an almost 80% reduction in the risk of death[2][3]. The most common side effects included infections, respiratory problems, seizures, vomiting, fever, anemia, and injection site reactions[4]. Because copper can accumulate in the body, patients receiving Zycubo require close monitoring for potential toxicity[4]. Physicians emphasize that early diagnosis and rapid initiation of treatment are critical to maximize survival benefits in affected neonates[5].