• Posted March 15, 2024

FDA Approves First Drug for Common, Serious Liver Disease

Millions of Americans whose livers develop scar tissue due to a common disease now have the first approved drug, Rezdiffra, to treat the condition, the U.S. Food and Drug Administration announced Thursday.

The condition is called non-cirrhotic non-alcoholic steatohepatitis (NASH). NASH with liver scarring is thought to affect up to 8 million Americans. It's often tied to high blood pressure and type 2 diabetes, the FDA explained in a statement.

“Previously, patients with NASH who also have notable liver scarring did not have a medication that could directly address their liver damage,” said Dr. Nikolay Nikolov, 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," Nokolov said in the FDA statement.

The liver is a vital organ that cleanses the blood and helps with digestion by secreting bile. However, over time, inflammation caused by obesity, diabetes and high blood pressure can cause nonalcoholic liver disease, which triggers fat accumulation in the liver, liver scarring and dysfunction.

As the FDA explained, "Rezdiffra is a partial activator of a thyroid hormone receptor; activation of this receptor by Rezdiffra in the liver reduces liver fat accumulation."

The agency said that the drug was approved under an "accelerated" pathway because it addressed "an unmet medical need."

Rezdiffra is made by Madrigal Pharmaceuticals and was approved based on data collected after a year as part of an ongoing 54-month trial. That data assessed improvement in levels of liver scarring compared to patients who were taking a placebo.

Based on the assessments of various pathologists who were examining patients' liver scarring, between 24% and 36% of NASH patients taking 100 milligrams of Rezdiffra saw their NASH resolve and experienced no worsening of liver scarring, compared to 9% to 13% of those who received placebo, the FDA said. A similar ratio saw their NASH continuing but experienced no worsening of liver scarring, the agency said.

Results like these within one year is "notable," the FDA said, since "the disease typically progresses slowly, with a majority of patients taking years or even decades to show progression."

Rezdiffra did come with side effects, including diarrhea and nausea, and its label includes warnings regarding a risk for drug-induced liver toxicity and gallbladder issues.

Rezdiffra shouldn't be taken by patients with a more advanced form of liver disease called decompensated cirrhosis, the FDA said, and the drug may interact with cholesterol-lowering statins, so patients should talk over the use of Rezdiffra with their doctor.

More information

Find out more about NASH at Stanford Medicine.

SOURCE: U.S. Food and Drug Administration, news release, March 14, 2024