Overview
- The FDA cleared the copper histidinate injection for children with the X-linked disorder, marking the first authorized therapy in the United States.
- Zycubo (CUTX-101) is administered subcutaneously to bypass gastrointestinal absorption and raise blood copper levels.
- Supporting data showed a significant survival benefit with early treatment, with median overall survival of 177.1 months versus 17.6 months in an untreated external control group.
- Reported adverse reactions included infections, fever, hemorrhage, low blood pressure, respiratory failure, vomiting, diarrhea, fractures, elevated transaminases, anemia, fungal infections, and injection-site reactions.
- Commercial terms grant Fortress/Cyprium tiered royalties and up to $129 million in milestones from Sentynl, a priority review voucher will be transferred to Cyprium, and Fortress shares rose more than 13% in premarket trading.