The FDA has granted approvals expanding treatment options for rare diseases and cancers. Saga Diagnostics secured Medicare coverage for its Pathlight MRD test monitoring breast cancer recurrence with high sensitivity. Apellis Pharmaceuticals’ pegcetacoplan (Empaveli) gained approval for two rare kidney diseases, while PTC Therapeutics’ oral sepiapterin (Sephience) was authorized for phenylketonuria treatment in adults and children. Concurrently, collaboration initiatives and trial advancements continue in oncology, including MD Anderson and Toppan Holdings' organoid assay development for personalized cancer therapy.