The FDA issued a complete response letter rejecting the NDA for CUTX-101, a copper replacement therapy for rare pediatric Menkes disease, citing manufacturing facility deficiencies. This setback occurred despite lack of efficacy or safety concerns. Additionally, regulatory guidance finalized for the Inflation Reduction Act preserves orphan drug research incentives, improving transparency in Medicare drug price negotiations. These developments reflect ongoing regulatory challenges and policy adjustments influencing rare disease therapeutics.