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GENFIT Receives FDA Orphan Drug Designation for NTZ for the treatment of ACLF
FDA grants Orphan Drug Designation (ODD) for NTZ for ACLF, a severe condition with no approved therapiesNTZ is being advanced in ACLF through its G1090N reformulation, designed to unlock its clinical potential for patients facing this life‑threatening conditionThis regulatory milestone follows favorable Phase 1 safety results and strong anti-inflammatory activity observed in ex vivo assays on samples from healthy volunteers and cirrhotic donors Lille (France), Cambridge (Massachusetts, United St
About this update from Genfit Sa
[{"type":"list","items":[{"val":[{"type":"text","content":"FDA grants Orphan Drug Designation (ODD) for NTZ for ACLF, a severe condition with no approved therapies","length":104,"tagName":"p"}]},{"val":[{"type":"text","content":"NTZ is being advanced in ACLF through its G1090N reformulation, designed to unlock its clinical potential for patients facing this life‑threatening condition","length":157,"tagName":"p"}]},{"val":[{"type":"text","content":"This regulatory milestone follows favorable Phase 1 safety results and strong anti-inflammatory activity observed in ex vivo assays on samples from healthy volunteers and cirrhotic donors","length":187,"tagName":"p"}]}],"tagName":"ul","bulletedList":true,"length":448,"olType":false},{"type":"text","content":"Lille (France), Cambridge (Massachusetts, United States), Zurich (Switzerland), March 9, 2026 - GENFIT (Euronext: GNFT), a biopharmaceutical company dedicated to improving the lives of patients with rare and life-threatening liver diseases, today announces that the U.S. Food and Drug Administration (FDA) has granted ODD to NTZ (nitazoxanide), its investigational small-molecule drug candidate developed as a new formulation for the treatment of Acute-on-Chronic Liver Failure (ACLF).","length":485,"tagName":"p"},{"type":"text","content":"G1090N is GENFIT’s lead investigational program within the ACLF segment of its pipeline. The FDA’s ODD recognizes the potential of G1090N’s active substance to address this severe, rare condition characterized by rapid deterioration, systemic inflammation, and high short-term mortality.","length":287,"tagName":"p"},{"type":"text","content":"This designation follows recent Phase 1 data demonstrating a favorable safety and tolerability profile in healthy volunteers, as well as compelling anti-inflammatory activity across ex vivo models, providing a solid foundation for advancing the program toward initiation of Phase 2 clinical development, targeted for the second half of 2026. ODD also provides development incentives, including FDA regulatory guidance, certain user fee reductions, and eligibility for seven-year U.S. market exclusivity for the designated indication upon FDA approval.","length":551,"tagName":"p"},{"type":"text","content":"ENDABOUT GENFIT","length":16,"tagName":"p"},{"type":"text","content":"GENFIT is a biopharmaceutical company committed to improving the lives of ...