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Background <p>Efruxifermin, a long-acting FGF21 analog, has shown promising results in adults with metabolic dysfunction-associated steatohepatitis (MASH). We conducted a meta-analysis to evaluate the efficacy and safety of efruxifermin in biopsy-confirmed MASH/NASH.</p> Methods <p>We searched PubMed, Cochrane Library, Scopus, and Web of Science from inception to 5 June 2025 for randomized controlled trials (RCTs)&#xa0;comparing efruxifermin with placebo. Random-effects models pooled mean differences (MDs)&#xa0;for continuous outcomes and risk ratios (RRs)&#xa0;for binary outcomes with 95% confidence intervals&#xa0;(CIs). Trial sequential analysis (TSA) evaluated whether the accumulated evidence was sufficient for firm conclusions.</p> Results <p>Four RCTs (<i>n</i> = 419&#xa0;patients) were included. Efruxifermin improved ≥ 1-stage fibrosis without MASH worsening (39.7% vs. 17.1%, RR 1.83, 95%CI: [1.17–2.84]) and MASH resolution without worsening (45.1% vs. 13.5%, RR 2.42, 95% CI: [1.48–3.94]). However, TSA suggested the evidence is not yet conclusive. Efruxifermin also reduced ALT (MD −13.97, 95% CI [−23.12; −4.82]), AST (MD −13.24, 95% CI [−21.29; −5.20]), and ELF score (MD −0.66, 95% CI [−0.84; −0.49]). However, no benefit was seen for ≥ 2-stage fibrosis (<i>p</i> = 0.25). Drug-related adverse events (AEs) (71.2% vs. 43.7%%, RR 1.35, 95% CI [1.06–1.71]) and discontinuations (10.3% vs. 2.2%, RR 3.13, 95% CI [1.12–8.76]) increased, but not serious AEs or mortality.</p> Conclusions <p>Efruxifermin may produce meaningful histologic and biomarker improvements in MASH, but tolerability may limit persistence. Larger phase III RCTs with longer follow-up are needed to confirm durability, clarify effects in cirrhosis, and better define safety.</p>

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Clinical and metabolic benefits of efruxifermin in MASH: a GRADE-assessed meta-analysis of randomized trials with trial sequential analysis

  • 期刊论文

  • 发表日期：

    2026年3月17日

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  • Islam Rajab,
  • Ahmed Emara,
  • Mohamed S. Elgendy,
  • Ameer Awashra,
  • Mohamed R. Ezz,
  • Eman Ayman Nada,
  • Furqan A. Bhullar,
  • Yana Cavanagh,
  • Walid Baddoura

摘要

Background

Efruxifermin, a long-acting FGF21 analog, has shown promising results in adults with metabolic dysfunction-associated steatohepatitis (MASH). We conducted a meta-analysis to evaluate the efficacy and safety of efruxifermin in biopsy-confirmed MASH/NASH.

Methods

We searched PubMed, Cochrane Library, Scopus, and Web of Science from inception to 5 June 2025 for randomized controlled trials (RCTs) comparing efruxifermin with placebo. Random-effects models pooled mean differences (MDs) for continuous outcomes and risk ratios (RRs) for binary outcomes with 95% confidence intervals (CIs). Trial sequential analysis (TSA) evaluated whether the accumulated evidence was sufficient for firm conclusions.

Results

Four RCTs (n = 419 patients) were included. Efruxifermin improved ≥ 1-stage fibrosis without MASH worsening (39.7% vs. 17.1%, RR 1.83, 95%CI: [1.17–2.84]) and MASH resolution without worsening (45.1% vs. 13.5%, RR 2.42, 95% CI: [1.48–3.94]). However, TSA suggested the evidence is not yet conclusive. Efruxifermin also reduced ALT (MD −13.97, 95% CI [−23.12; −4.82]), AST (MD −13.24, 95% CI [−21.29; −5.20]), and ELF score (MD −0.66, 95% CI [−0.84; −0.49]). However, no benefit was seen for ≥ 2-stage fibrosis (p = 0.25). Drug-related adverse events (AEs) (71.2% vs. 43.7%%, RR 1.35, 95% CI [1.06–1.71]) and discontinuations (10.3% vs. 2.2%, RR 3.13, 95% CI [1.12–8.76]) increased, but not serious AEs or mortality.

Conclusions

Efruxifermin may produce meaningful histologic and biomarker improvements in MASH, but tolerability may limit persistence. Larger phase III RCTs with longer follow-up are needed to confirm durability, clarify effects in cirrhosis, and better define safety.