Genfit announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to its investigational small‑molecule NTZ, a new formulation of nitazoxanide (G1090N), for the treatment of Acute‑on‑Chronic Liver Failure (ACLF). The designation provides Genfit with development incentives, including regulatory guidance, user‑fee reductions, and a seven‑year U.S. market exclusivity period if the drug is approved for ACLF.
The decision follows Phase 1 data that demonstrated a favorable safety and tolerability profile in healthy volunteers and subjects with hepatic impairment, as well as anti‑inflammatory activity in ex‑vivo models. Genfit plans to advance NTZ into Phase 2 clinical development, targeted for the second half of 2026, after a Phase 2a proof‑of‑concept study was previously discussed with the FDA for the second half of 2023 and a Phase 2 trial for G1090N was anticipated to begin in the first quarter of 2025.
ACLF is a severe syndrome with high short‑term mortality and no approved specific therapies. The prevalence of ACLF was estimated at approximately 294,000 patients in 2021 across the U.S., EU4, and the U.K., with projections of about 300,000 by 2036. Genfit’s focus on ACLF follows the discontinuation of its VS‑01 program in this indication, making NTZ a critical asset in the company’s liver‑disease portfolio.
Genfit’s financial position supports continued investment in the ACLF program. The company reported €65.4 million in revenue for 2025, down from €67.0 million in 2024, and held €101.1 million in cash and cash equivalents as of December 31 2025. The orphan designation is expected to de‑risk the pipeline and potentially unlock a sizable market opportunity for a condition with a clear unmet need.
Dr. Carol Addy, Genfit’s Chief Medical Officer, said, "We are pleased with the encouraging data and the outcome of this Phase 1 study, which supports the potential of NTZ in ACLF and the rationale for further exploration of this drug in this indication. ACLF is an underserved medical condition given its high short‑term mortality and the fact that no drugs are currently approved for this indication."
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