April 27, 2026
1 min read
Key takeaways:
- Phase 2 trial data show fibrosis improvement and MASH resolution with efimosfermin compared with placebo.
- Multiple phase 3 trials are underway or in development.
The FDA granted breakthrough therapy designation to efimosfermin — an investigational, once-monthly fibroblast growth factor 21 analogue — for treatment of patients with metabolic dysfunction-associated steatohepatitis.
According to a manufacturer press release, efimosfermin (GSK) is a liver therapy “designed to regulate key metabolic pathways to decrease liver fat, ameliorate liver inflammation and reverse liver fibrosis in patients with MASH.”
The FDA granted breakthrough therapy designation to efimosfermin — an investigational, once-monthly fibroblast growth factor 21 analogue — for treatment of patients with metabolic dysfunction-associated steatohepatitis.
“MASH affects millions of people worldwide and is one of the leading causes of liver transplant in the U.S. and Europe, but treatment options are limited for most and nonexistent for those with the most advanced form of disease,” Kaivan Khavandi, MD, PhD, senior vice president and global head of translational and development sciences at GSK, said in the release.
“We believe efimosfermin has the potential to significantly advance the standard of care by directly targeting liver fibrosis.”
The FDA based its decision on data from a randomized, phase 2 trial of patients with MASH with moderate to advanced and cirrhotic fibrosis.
As Healio previously reported, results of the trial showed a higher proportion of patients who received efimosfermin vs. placebo had improvement in fibrosis by at least one stage without worsening of MASH (45.2% vs. 20.6%; P = .038).
A greater proportion of patients who received efimosfermin also achieved MASH resolution without worsening of fibrosis (67.7% vs. 29.4%; P = .002).
According to the release, the most common adverse events included nausea, diarrhea and vomiting, which were mild and transient.
The ZENITH-1 and ZENITH-2 phase 3 trials are currently underway to assess efficacy and safety of efimosfermin among patients with MASH with F2/F3 fibrosis. A phase 3 trial that includes patients with F4 fibrosis is expected to begin this year.
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