First drug for NASH with liver fibrosis submitted to FDA

Non-alcoholic fatty liver disease

Madrigal Pharmaceuticals has submitted an application for resmetirom, a drug for nonalcoholic steatohepatitis (NASH) with liver fibrosis, to the US Food and Drug Administration (FDA) and has requested a priority review.

Resmetirom is a once daily, oral, thyroid hormone receptor (THR)-β selective agonist designed to target key underlying causes of NASH in the liver.

The clinical development programme for resmetirom is comprised of 18 clinical studies supporting the NDA: twelve Phase I studies, two Phase II studies, and four Phase III studies.

In the pivotal Phase III MAESTRO-NASH serial liver biopsy trial, resmetirom achieved both liver histological improvement endpoints – resolution of NASH and reduction of liver fibrosis – proposed by FDA as reasonably likely to predict clinical benefit to support accelerated approval for the treatment of NASH with liver fibrosis.

In both MAESTRO-NASH and the Phase III safety study MAESTRO-NAFLD-1, atherogenic lipids and lipoproteins, including LDL cholesterol and triglycerides, were reduced by resmetirom and patients achieved potentially meaningful improvements in noninvasive measures of liver health.

In April 2023, resmetirom was granted Breakthrough Therapy designation by FDA for the treatment of adults with NASH with liver fibrosis.

Becky Taub, Chief Medical Officer and President of Research & Development of Madrigal, stated: “Based on the positive efficacy results observed in MAESTRO-NASH and the large safety database we have established across the MAESTRO trials, we believe resmetirom has the potential to become the first approved treatment for patients with NASH with liver fibrosis, a disease that is rapidly becoming the leading cause of liver transplantation in the US.”

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