U.S. FDA approves Fortress Bio and Zydus treatment for Menkes disease

The U.S. Food and Drug Administration approved Zycubo, developed by Fortress Biotech and Zydus Lifesciences, as the first U.S. treatment for Menkes disease. The drug is an under-the-skin injectable form of copper histidinate and data reported showed a large survival benefit with early treatment.

The U.S. Food and Drug Administration has approved Zycubo, a treatment from Fortress Biotech and Zydus Lifesciences, for children with Menkes disease. Menkes disease is a rare genetic disorder that prevents the body from absorbing copper from the diet. The approval makes Zycubo the first drug on the U.S. market specifically for this condition. Companies have reported that the disease primarily affects male infants and provided recent prevalence estimates. Key facts: - Zycubo, chemically known as CUTX-101, is an under-the-skin injectable formulation of copper histidinate designed to raise blood copper levels. - The article reports that approval was based on data showing early treatment improved overall survival, with a nearly 80% reduction in the risk of death compared with untreated patients. - Symptoms of Menkes disease include sparse, depigmented hair, seizures and developmental delays, and many untreated patients die between about ages two and three. - The companies cited prevalence estimates ranging from about 1 in 34,810 to 1 in 8,664 live male births, and the disease primarily affects male infants. - The article reports that Fortress is eligible for tiered royalties on Zycubo sales and up to $129 million in aggregate development and sales milestones from Zydus, and that Fortress shares rose over 13% in premarket trading. Summary: The approval provides the first U.S. approved therapy for Menkes disease. Undetermined at this time.