FDA Approves First Drug to Treat Serious Liver Disease — A Win for Madrigal Pharmaceuticals as Rivals Circle

By Trisha Andrada

Mar 15 2024, 08:12 AM EDT

The US Food and Drug Administration (FDA) announced Thursday that it had approved the first drug to treat serious liver disease. In a press release, the FDA said it approved Rezdiffra (resmetirom) for the treatment of adults with a type of liver inflammation called noncirrhotic non-alcoholic steatohepatitis (NASH).

According to The Hill, NASH, also known as metabolic dysfunction-associated steatohepatitis (MASH), occurs when the liver becomes inflamed or damaged due to the buildup of fat in the liver.

This liver inflammation can eventually lead to liver scarring and liver dysfunction. The FDA said NASH is often linked to type 2 diabetes and high blood pressure.

"Previously, patients with NASH who also have notable liver scarring did not have a medication that could directly address their liver damage," Nikolay Nikolov, MD, acting director of the Office of Immunology and Inflammation in the FDA's Center for Drug Evaluation and Research, said in the statement.

"Today's approval of Rezdiffra will, for the first time, provide a treatment option for these patients, in addition to diet and exercise," he added.

Success of Madrigal Pharmaceuticals For First Treatment Drug for NASH Liver Disease

Rezdiffra was developed by the biotechnology company Madrigal Pharmaceuticals. With the FDA's decision, the company has succeeded in a disease area that several larger firms have failed or are still attempting to break into.

According to CNBC, the popular weight reduction injections made by Novo Nordisk and Eli Lilly are both being tested as potential treatments for the same kind of liver disease. Madrigal shares soared over 20% in extended trading Thursday following the FDA's approval.

About the Rezdiffra Drug

According to the FDA, around six to eight million people in the US have NASH with moderate to advanced liver scarring. The agency said Rezdiffra is a partial activator of a thyroid hormone receptor, and "activation of this receptor by Rezdiffra in the liver reduces liver fat accumulation."

The FDA noted that the most common side effects of Rezdiffra included diarrhea and nausea, and it comes with warnings on drug-induced liver toxicity and gallbladder-related side effects.

The agency said Rezdiffra should be avoided in patients with decompensated cirrhosis and "patients should stop using Rezdiffra if they develop signs or symptoms of worsening liver function."

The FDA added that using Rezdiffra with other drugs, particularly statins for lowering cholesterol, may result in potentially significant drug interactions.