FDA approves first MASH drug

The first drug to treat fatty liver disease due to metabolic dysfunction-associated steatohepatitis (MASH) has been given a green light by the US Food and Drug Administration. Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) received an accelerated approval to treat the disease, previously known as non-alcoholic steatohepatitis (NASH). In this progressive liver condition fat buildup triggers inflammation and cell damage, leading to fibrosis and eventually cirrhosis. The complex pathophysiology of the condition and need for long-term safety have until now hindered drug discovery efforts. Rezdiffra is an oral thyroid hormone receptor-β (THR-β) agonist that improves lipid metabolism and mitochondrial activity in liver cells.

The FDA’s go-ahead was based in part on a phase 3 trial published in the New England Journal of Medicine. Biopsy samples from over 900 people showed that fat buildup and inflammation cleared without worsening fibrosis in 25% of patients at 12 months, compared with approximately 10% on placebo. In addition, fibrosis improved without worsening fatty liver symptoms in around 25% of patients, compared with 14% of patients in the placebo group. The drug also improved low-density lipoprotein cholesterol levels.

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