Global pharma powerhouse GSK has secured a major regulatory lift for its experimental liver therapy efimosfermin, after the US FDA granted it Breakthrough Therapy Designation and the European Medicines Agency awarded it Priority Medicines status for the treatment of MASH.

 

The twin designations are designed to speed up the development and review of promising medicines targeting serious conditions with high unmet need.

 

Kaivan Khavandi, SVP, R&D Head Respiratory, Immunology & Inflammation (RI&I), GSK, said: “MASH affects millions of people worldwide and is one of the leading causes of liver transplant in the US and Europe, but treatment options are limited for most and non-existent for those with the most advanced form of disease.

 

"These designations recognise efimosfermin’s potential and reflect GSK’s accelerating momentum in liver health. We believe efimosfermin has the potential to significantly advance the standard of care by directly targeting liver fibrosis.”

 

The regulatory momentum is backed by clinical data in patients with moderate-to-advanced and cirrhotic fibrosis. In a phase II study, F2/F3 patients treated for 48 weeks showed fibrosis improvement and MASH resolution compared with placebo. The therapy also demonstrated a generally well-tolerated safety profile, with mostly mild and transient side effects including nausea, vomiting and diarrhoea.

 

Efimosfermin is now advancing through phase III development, with the ZENITH-1 and ZENITH-2 trials evaluating its efficacy and safety in F2/F3 MASH patients. Additional phase III studies in cirrhotic patients are expected to begin later this year.