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

Core Viewpoint - The FDA has set a new PDUFA target action date of January 14, 2026, for the resubmission of the New Drug Application (NDA) for CUTX-101, a treatment for Menkes disease in pediatric patients [1][2]. Group 1: FDA Resubmission and Approval Process - The resubmission of the NDA for CUTX-101 has been accepted as a Class 1 resubmission, leading to the new PDUFA target action date [2]. - Sentynl Therapeutics, Inc. has taken full responsibility for the development and commercialization of CUTX-101 from Cyprium, following a complete response letter (CRL) from the FDA that did not raise concerns about the drug's efficacy and safety [3][5]. - The CRL issued on September 30, 2025, cited issues related to the manufacturing site's cGMP compliance but did not identify any deficiencies in the clinical data supporting CUTX-101 [3]. Group 2: Financial and Developmental Aspects - Upon approval, Sentynl will transfer a Rare Pediatric Disease Priority Review Voucher (PRV) to Cyprium and Cyprium will be eligible for royalties on net sales of CUTX-101, along with up to $129 million in development and sales milestones [4]. - The NDA for CUTX-101 was initially granted Priority Review by the FDA, supported by positive clinical efficacy results demonstrating significant improvement in overall survival for patients with Menkes disease [5]. Group 3: Menkes Disease Overview - Menkes disease is a rare X-linked recessive pediatric condition caused by mutations in the copper transporter ATP7A, with a birth prevalence estimated between 1 in 34,810 and 1 in 8,664 live male births [6]. - The disease is characterized by severe neurological symptoms and high mortality rates in untreated cases, with many patients not surviving past the age of 2-3 years [6]. Group 4: Company Background - Cyprium Therapeutics focuses on developing therapies for Menkes disease and related copper metabolism disorders, having entered into a Cooperative Research and Development Agreement with the NIH to advance CUTX-101 [7]. - Fortress Biotech, the parent company of Cyprium, is an innovative biopharmaceutical firm with a diverse portfolio, including eight marketed products and multiple development programs across various therapeutic areas [8][9].