FDA Issues Complete Response Letter to Fortress Biotech's Menkes Disease Drug CUTX-101 Due to Manufacturing Deficiencies

Fortress Biotech and its subsidiary Cyprium Therapeutics received a Complete Response Letter (CRL) from the FDA regarding their New Drug Application for CUTX-101, a treatment for Menkes disease in pediatric patients. The FDA's CRL highlighted deficiencies in the manufacturing practices at the facility producing CUTX-101 but raised no concerns about the drug's efficacy or safety. Sentynl Therapeutics, which took over development and commercialization of CUTX-101 in late 2023, is expected to address the FDA's concerns and resubmit the NDA promptly. The CRL follows a recent FDA re-inspection of the manufacturing facility, and Sentynl plans to request a meeting with the FDA to discuss next steps. Upon approval, Cyprium will receive a Rare Pediatric Disease Priority Review Voucher from Sentynl and could earn royalties and up to $129 million in milestone payments. Menkes disease is a rare genetic pediatric disorder, and CUTX-101 showed promising clinical results demonstrating improved survival with early treatment.