GSK’s efimosfermin receives FDA breakthrough therapy and EMA priority medicines designations for MASH

GSK has recently announced that efimosfermin, its once-monthly investigational liver therapy, has received Breakthrough Therapy Designation from the FDA and Priority Medicines (PRIME) Designation from the European Medicines Agency for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). Both designations recognise the therapy’s potential to address a significant unmet medical need.

MASH is a chronic, progressive liver disease affecting up to 5% of the global population and is a leading cause of liver transplants in both the US and Europe. The buildup of scar tissue, or fibrosis, is a key predictor of serious outcomes including cirrhosis, liver failure and liver cancer. Treatment options are currently limited for patients with moderate to advanced fibrosis, and there are no approved treatments at all for those with cirrhotic MASH.

The designations were supported by Phase II data in patients with moderate to advanced fibrosis, which showed that once-monthly efimosfermin improved liver fibrosis and achieved MASH resolution compared to placebo at 48 weeks, with a well-tolerated safety profile. Mild transient adverse events including nausea, vomiting and diarrhoea were reported.

Kaivan Khavandi, GSK’s R&D Head for Respiratory, Immunology and Inflammation, said the results point to a meaningful step forward for patients with few options: “We believe efimosfermin has the potential to significantly advance the standard of care by directly targeting liver fibrosis. These designations recognise efimosfermin’s potential and reflect GSK’s accelerating momentum in liver health.”

Efimosfermin is currently in Phase III through the ZENITH-1 and ZENITH-2 trials in patients with moderate to advanced fibrosis, with Phase III trials in cirrhotic MASH patients expected to begin this year.

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