Fortress Biotech, Inc. and its majority‑owned subsidiary Cyprium Therapeutics announced that the U.S. Food and Drug Administration has accepted the resubmission of the New Drug Application for CUTX‑101, a copper histidinate therapy for Menkes disease. The acceptance, classified as a Class 1 resubmission, sets a new Prescription Drug User Fee Act (PDUFA) target action date of January 14 2026. The decision follows a Complete Response Letter issued on September 30 2025 that cited manufacturing site cGMP compliance issues but did not raise concerns about the drug’s efficacy or safety data.
CUTX‑101 is the first therapy under development for Menkes disease, a rare X‑linked recessive disorder that causes fatal neurodegeneration in infants. Sentynl Therapeutics, a wholly‑owned subsidiary of Zydus Lifesciences, assumed full responsibility for the program in December 2023 and resubmitted the revised NDA on November 14 2025. The FDA’s acceptance indicates that the manufacturing deficiencies identified in the September CRL have been adequately addressed, allowing the review to proceed on a streamlined schedule.
The regulatory milestone unlocks several financial opportunities for Fortress. Upon eventual FDA approval, Cyprium will receive a Rare Pediatric Disease Priority Review Voucher that can be sold for an estimated $100 million to $150 million or more, or used to accelerate other product approvals. In addition, Cyprium is eligible for up to $129 million in development and sales milestones from Sentynl, and Fortress stands to earn royalties on net sales of the therapy. The combination of a high‑value voucher, milestone payments, and royalty upside represents a potentially significant non‑dilutive cash infusion that could fund future pipeline development.
Matt Heck, CEO of Sentynl, said the FDA’s acceptance “brings us one step closer to a milestone for patients and families who are living with Menkes disease.” The approval also validates Fortress’s diversified rare‑disease strategy, demonstrating that its pipeline can navigate regulatory hurdles and reach the market. The company’s management has highlighted the importance of this event as a proof point for its acquisition‑and‑development model and a catalyst for future deals.
The Menkes disease market remains unserved, with no FDA‑approved treatments available. CUTX‑101’s potential first‑in‑class status, combined with the PRV’s high market value, positions Fortress to capture early market share if the therapy is approved. The January 2026 PDUFA target places the drug in the same window as other high‑profile pediatric approvals, offering a strategic advantage in a crowded regulatory landscape. Overall, the FDA acceptance signals a critical de‑risking of the program and a tangible path to revenue generation for Fortress and its partners.
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