GENFIT’s NTZ Receives FDA Orphan Drug Designation for Acute-on-Chronic Liver Failure

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to GENFIT’s investigational drug, nitazoxanide (NTZ), for the treatment of Acute-on-Chronic Liver Failure (ACLF), a severe and rare condition with limited treatment options.

Understanding the Significance of the Designation

The ODD recognizes the potential of NTZ, specifically its G1090N reformulation, to address the critical unmet medical need in ACLF. ACLF is characterized by rapid deterioration of liver function, systemic inflammation and a high risk of short-term mortality. The FDA’s ODD program provides several benefits to companies developing drugs for rare diseases, including regulatory guidance, potential fee reductions, and eligibility for seven years of market exclusivity in the U.S. Upon approval.

About G1090N and GENFIT’s Approach

GENFIT is advancing NTZ through a novel formulation, G1090N, designed to unlock its clinical potential for patients with ACLF. This reformulation is the cornerstone of GENFIT’s ACLF pipeline. Recent Phase 1 clinical trials have demonstrated a favorable safety and tolerability profile for G1090N in healthy volunteers. Preclinical studies have shown compelling anti-inflammatory activity in ex vivo models using samples from both healthy volunteers and patients with cirrhosis.

What is Acute-on-Chronic Liver Failure (ACLF)?

ACLF represents a severe and often life-threatening complication of chronic liver disease. It is defined by the sudden worsening of liver function in patients with underlying chronic liver conditions, leading to organ failure and a high mortality rate.

Looking Ahead: Phase 2 Clinical Development

GENFIT is planning to initiate Phase 2 clinical development of G1090N in the second half of 2026. This next phase of trials will be crucial in evaluating the efficacy of G1090N in patients with ACLF.

About GENFIT

GENFIT is a biopharmaceutical company dedicated to developing therapies for rare and life-threatening liver diseases. The company has a strong scientific heritage and a portfolio of investigational drugs targeting various liver conditions, including ACLF, acute decompensation in cirrhosis, and hepatic encephalopathy. GENFIT’s expertise was demonstrated by the accelerated approval of Iqirvo® (elafibranor) for Primary Biliary Cholangitis (PBC) in the US, Europe, and the UK. GENFIT is also involved in diagnostic solutions, such as NIS2+, for detecting metabolic dysfunction-associated steatohepatitis (MASH).