GlobeNewswire·Dec 15, 2025, 8:00 AM ET

Fortress Biotech and Cyprium Therapeutics Announce FDA Acceptance of CUTX-101 NDA Resubmission

The FDA accepted the Class 1 resubmission of the New Drug Application for CUTX-101 (copper histidinate) to treat Menkes disease in pediatric patients.
The new Prescription Drug User Fee Act target action date for the NDA is set for January 14, 2026.
Sentynl Therapeutics resubmitted the revised NDA on November 14, 2025, after addressing manufacturing site cGMP compliance issues raised in a September 30, 2025 Complete Response Letter.
The prior Complete Response Letter did not identify deficiencies in CUTX-101's efficacy or safety data, which show significant improvement in overall survival for early-treated Menkes disease subjects.
Sentynl will transfer the Rare Pediatric Disease Priority Review Voucher to Cyprium upon approval, while Cyprium will receive royalties on net sales plus up to $129 million in aggregate development & sales milestones.
Menkes disease is a rare X-linked recessive condition caused by ATP7A gene mutations, characterized by severe neurological symptoms & high mortality, with no currently FDA-approved treatments.
Cyprium Therapeutics, a majority-owned subsidiary of Fortress Biotech, holds a worldwide exclusive license for AAV-ATP7A gene therapy to be used in combination with CUTX-101, which is in pre-clinical development.
Fortress Biotech retains significant minority ownership in Sentynl Therapeutics & continues to advance its portfolio through partnerships & subsidiaries.