FDA approves first drug for serious liver disease

The U.S. Food and Drug Administration (FDA) has approved the first drug to treat serious liver disease, the agency announced in a release Thursday.

The FDA approved Rezdiffra (resmetirom) for the treatment of adults with a common form of liver inflammation called nonalcoholic steatohepatitis, or NASH.

NASH, also known as metabolic dysfunction-associated steatohepatitis, or MASH, happens when the liver becomes inflamed because of excessive fatty cells. Liver inflammation from NASH, over time, can lead to liver scarring and liver dysfunction.

Type 2 diabetes and high blood pressure are other health conditions often associated with NASH, according to the FDA.

“Previously, patients with NASH who also have notable liver scarring did not have a medication that could directly address their liver damage,” said Nikolay Nikolov, M.D., acting director of the Office of Immunology and Inflammation in the FDA’s Center for Drug Evaluation and Research. “Today’s approval of Rezdiffra will, for the first time, provide a treatment option for these patients, in addition to diet and exercise.” 

One estimate cited in the FDA’s release says approximately 6-8 million people in the U.S. have NASH with moderate to advanced liver scarring. Rezdiffra is a partial activator of the thyroid hormone receptor, and activation of this receptor by Rezdiffra in the liver “reduces liver fat accumulation,” according to the release.

Common side effects of Rezdiffra included diarrhea and nausea. In addition, warnings for the drug include drug-induced liver toxicity and gallbladder-related side effects. 

Patients with decompensated cirrhosis should not use Rezdiffra, according to the release. Meanwhile, patients should stop using the treatment if they develop signs or symptoms of worsening liver function.

 Using Rezdiffra with other drugs, in particular statins for lowering cholesterol, may result in potentially significant drug interactions, per the release.