FDA Conditionally Approves Madrigal Pharmaceuticals' Resmetirom As First Treatment For Fatty Liver Disease With Scarring

The FDA granted accelerated approval on Thursday to Madrigal Pharmaceuticals Inc.’s MDGL Rezdiffra (resmetirom) noncirrhotic nonalcoholic steatohepatitis (NASH) with moderate to advanced liver scarring (fibrosis) to be used along with diet and exercise. 

Continued approval for this indication may be contingent upon verification and description of clinical benefit in ongoing confirmatory trials.

This marks the first FDA approval of a treatment for patients with liver scarring due to fatty liver disease.

Rezdiffra is a once-daily, oral THR-β agonist designed to target key underlying causes of NASH. 

The accelerated approval of Rezdiffra was based on results from the Phase 3 MAESTRO-NASH trial that enrolled 1,759 patients.

Following 52 weeks of treatment, 100 mg and 80 mg doses of Rezdiffra demonstrated statistically significant improvement compared to placebo on two primary endpoints: NASH resolution with no worsening of fibrosis and an improvement in fibrosis by at least one stage with no worsening of the NAFLD activity score. 

At 12 months, liver biopsies showed 26%-27% of subjects on 80 mg and 24%-36% on 100 mg of Rezdiffra experienced NASH resolution and no worsening of liver scarring, compared to 9%-13% on placebo. 

Twenty-three percent of subjects on 80 mg and 24%-28% on 100 mg of Rezdiffra experienced an improvement in liver scarring and no worsening of NASH, compared to 13% to 15% on placebo.

The Rezdiffra prescribing information does not include a liver biopsy requirement for diagnosis. 

MAESTRO-NASH remains ongoing as an outcomes study designed to generate confirmatory data. 

A second ongoing outcomes trial is evaluating the progression to liver decompensation events in patients with well-compensated NASH cirrhosis treated with Rezdiffra versus placebo.

Rezdiffra is expected to be available to patients in the U.S. in April and will be distributed through a limited specialty pharmacy network. 

Approximately 6-8 million people in the U.S. have NASH with moderate to advanced liver scarring.

MDGL Price Action: Madrigal Pharmaceuticals shares are up 10.67% at $269.55 at last check Friday.

Read Next: Cannabis Stocks Hit The Roof As Hedge Funder Says Federal Descheduling Imminent

Photo: Julia Koblitz via Unsplash