Fortress Biotech's CUTX-101 New Drug Application Accepted by FDA
Fortress Biotech 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, intended to treat Menkes disease in pediatric patients. The resubmission has been accepted as a Class 1 resubmission and as a result, the new Prescription Drug User Fee Act target action date for the NDA is January 14, 2026. In December 2023, Sentynl Therapeutics assumed full responsibility for the development and commercialization of CUTX-101 from Cyprium. On November 14, 2025, Sentynl resubmitted the revised NDA after receiving a complete response letter from the FDA on September 30, 2025, which CRL cited observations regarding the manufacturing site's cGMP compliance. The CRL did not cite any other approvability concerns, nor did it identify any deficiencies in CUTX-101's efficacy and safety data, which demonstrate significant improvement in overall survival for Menkes disease subjects who received early treatment with the therapy. Pursuant to the transaction with Sentynl, Sentynl will transfer to Cyprium, if issued upon approval, a Rare Pediatric Disease Priority Review Voucher, and Cyprium will also be eligible to receive royalties on net sales of CUTX-101 and up to $129M in aggregate development and sales milestones from Sentynl. The CUTX-101 NDA was initially granted Priority Review by the FDA and is supported by positive topline clinical efficacy results for CUTX-101, demonstrating significant improvement in overall survival for Menkes disease subjects who received early treatment with CUTX-101.
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- FDA Approval: The U.S. FDA has officially approved ZYCUBO (formerly CUTX-101) for treating Menkes disease in children, marking it as the first FDA-approved treatment for this rare condition, significantly enhancing Fortress Biotech's market position in the rare disease sector.
- Milestone Earnings: Cyprium Therapeutics stands to earn tiered royalties and up to $129 million in potential development and sales milestones from Sentynl, which will substantially bolster the company's financial performance and support future R&D efforts.
- Survival Rate Improvement: Results indicate that children receiving early treatment with ZYCUBO had nearly an 80% lower risk of death, with a median overall survival of 177.1 months compared to 17.6 months in the control group, highlighting the drug's critical role in improving patient outcomes.
- Positive Market Reaction: Following the FDA approval, Fortress Biotech's stock surged over 12% in premarket trading, with investor sentiment shifting from 'bullish' to 'extremely bullish', reflecting strong market confidence and growth potential for the drug.
