Data Sheet 1_Efficacy and safety of efruxifermin for patients with NASH/MASH: an updated systematic review and meta-analysis.pdf

AimsEfruxifermin is a promising treatment for non-alcoholic steatohepatitis (NASH), now referred to as metabolic dysfunction-associated steatohepatitis (MASH). This meta-analysis aims to evaluate the efficacy and safety of efruxifermin in patients with NASH/MASH.
MethodsWe systematically searched PubMed, Embase, the Cochrane Library, and ClinicalTrials.gov for randomized controlled trials (RCTs) evaluating the efficacy and safety of efruxifermin in patients with NASH/MASH up to 6 August 2025. The primary outcomes were changes in liver fibrosis and steatosis, with safety assessed through adverse events.
ResultsThis meta-analysis included 4 RCTs with 419 participants. Compared with placebo, efruxifermin demonstrated a significant advantage in ≥1 stage improvement in liver fibrosis without worsening steatohepatitis (relative risk [RR]: 2.18, 95% confidence interval [CI] [1.34, 3.57], P = 0.002), NASH/MASH resolution with fibrosis improvement (RR: 5.15, 95% CI [1.52, 17.47], P = 0.009), and ≥2-point non-alcoholic fatty liver disease activity score (NAS) improvement without fibrosis worsening (RR: 3.34, 95% CI [1.93, 5.80], P < 0.001). Additionally, efruxifermin reduced the enhanced liver fibrosis (ELF) score, liver stiffness measurement (LSM), and serum levels of N-terminal type-III collagen pro-peptide (ProC3). For steatosis reduction, efruxifermin significantly increased the proportions of patients with ≥30% hepatic fat fraction (HFF) reduction (RR: 4.69, 95% CI [2.53, 8.71], P < 0.001), ≥50% HFF reduction (RR: 22.57, 95% CI [5.78, 88.22], P < 0.001), and liver fat normalization (RR: 13.03, 95% CI [3.30, 51.50], P < 0.001). However, efruxifermin treatment was associated with higher rates of both adverse events leading to discontinuation and gastrointestinal adverse events.
ConclusionEfruxifermin may represent a promising therapeutic option for NASH/MASH. Given the limitations in both the number and short follow-up duration of the included RCTs, the conclusions should be interpreted with caution. Further large-scale, multicenter, long-term, and high-quality RCTs are necessary to validate these results in diverse populations.
Systematic Review Registrationhttps://www.crd.york.ac.uk/PROSPERO/, identifier CRD42025111 4840.