UChicago Medicine will offer first-of-its-kind drug for metabolic liver disease

Liver disease specialists at the University of Chicago Medicine will soon begin prescribing a first-of-its-kind drug for treating advanced metabolic dysfunction-associated steatotic liver disease (MASLD) — formerly known as nonalcoholic fatty liver disease (NAFLD).

Resmetirom (to be sold under the brand name Rezdiffra), received FDA approval on March 14, 2024. It is the first medication approved for treating metabolic dysfunction-associated steatohepatitis (MASH), a more advanced stage of MASLD characterized by liver inflammation and scarring known as fibrosis.

“Until now, liver disease has never had a treatment shown to reverse fibrosis, which is the damage from which all other issues stem,” said Michael Charlton, MBBS, Director of the Transplant Institute at UChicago Medicine.

“If we can stop or slow fibrosis, we can theoretically prevent a lot of downstream consequences such as deaths, liver failure, liver cancer and the need for transplantation,” said Mary Rinella, MD, Director of Metabolic and Fatty/Steatotic Liver Disease at UChicago Medicine.

Along with research colleagues from UChicago Medicine and other health institutions, Charlton and Rinella advised and contributed to a phase three clinical trial of resmetirom that reported positive results in February 2024. After a year of treatment, a majority of trial participants who took resmetirom pills saw their liver fibrosis either reversed or halted in its progression.

“We succeeded in something people didn't think could be done for this disease: we reached a biological endpoint,” Charlton said. Biological endpoints are measurable health markers that regulators expect to be predictive of a treatment’s clinical benefit.

Rinella, Charlton and their collaborators are continuing their research even as the drug enters the clinic, extending the phase three trial to examine long-term outcomes. They emphasize that routine healthcare and screenings should focus more intentionally on diagnosing patients with MASH to maximize the impact of this breakthrough.

“We estimate that at least 25 million people in the United States could benefit from this drug, but only a small percentage of those have been identified in the clinic,” Charlton said.

Once supplies become available for clinical use, specialists at UChicago Medicine will begin incorporating resmetirom into treatment plans for eligible patients as part of their comprehensive approach to metabolic liver disease treatment that also includes dietary therapy and lifestyle modification.