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.
Access options
Access Nature and 54 other Nature Portfolio journals
Get Nature+, our best-value online-access subscription
24,99 € / 30 days
cancel any time
Subscribe to this journal
Receive 12 print issues and online access
209,00 € per year
only 17,42 € per issue
Buy this article
Purchase on Springer LinkInstant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
Additional access options:
Log in
Learn about institutional subscriptions
Read our FAQs
Contact customer support
About this article
Cite this article
FDA approves first MASH drug.
Nat Biotechnol 42, 540 (2024). https://doi.org/10.1038/s41587-024-02220-4
Download citation
Published: 17 April 2024
Issue Date: April 2024
DOI: https://doi.org/10.1038/s41587-024-02220-4
>>> Read full article>>>
Copyright for syndicated content belongs to the linked Source : Nature.com – https://www.nature.com/articles/s41587-024-02220-4
