GSK (LSE:GSK) announced efimosfermin, a once-monthly investigational liver therapy, has received Breakthrough Therapy Designation from the US Food and Drug Administration and Priority Medicines (PRIME) designation from the European Medicines Agency for the treatment of metabolic dysfunction-associated steatohepatitis (MASH).
The designations were supported by 48-week Phase II data in patients with moderate to advanced (F2/F3) fibrosis showing fibrosis improvement and MASH resolution versus placebo, and they recognise the potential to address a significant unmet medical need given MASH is a leading cause of liver transplant in the US and Europe.
"These designations recognise efimosfermin's potential and reflect GSK's accelerating momentum in liver health," said Kaivan Khavandi, SVP, R&D Head Respiratory, Immunology & Inflammation and Head of Translational & Development Sciences at GSK.
Efimosfermin is an investigational, once-monthly subcutaneous injection of a long-acting variant of FGF21 designed to reduce liver fat, ameliorate inflammation and reverse fibrosis, and it is not approved for use anywhere.
Phase II data also showed a generally well-tolerated safety profile with mild, transient adverse events such as nausea, vomiting and diarrhoea, and GSK said Phase III trials ZENITH-1 and ZENITH-2 in F2/F3 patients are under way with trials in F4 (cirrhotic) patients expected to start this year.
