The U.S. Food and Drug Administration (FDA) has approved a medication called Rezdiffra (resmetirom) for adults with noncirrhotic non-alcoholic steatohepatitis (NASH) with moderate to advanced liver scarring (fibrosis).
Nikolay Nikolov, M.D., acting director of the Office of Immunology and Inflammation in the FDA’s Center for Drug Evaluation and Research, touts the drug as the first that will address liver damage in patients with NASH. It works as a “partial activator of a thyroid hormone receptor; activation of this receptor by Rezdiffra in the liver reduces liver fat accumulation.”
NASH itself is a result of the “progression of nonalcoholic fatty liver disease where liver inflammation, over time, can lead to liver scarring and liver dysfunction. NASH is often associated with other health problems such as high blood pressure and type 2 diabetes. By at least one estimate, approximately 6-8 million people in the U.S. have NASH with moderate to advanced liver scarring, with that number expected to increase.”
Rezdiffra’s safety and efficacy was evaluated “based on an analysis of a surrogate endpoint at month 12 in a 54-month” study. The study is still ongoing. Patients involved in the study needed to have a “liver biopsy showing inflammation due to NASH with moderate or advanced liver scarring.” 888 subjects were then assigned to three groups – 294 received placebo, 298 received 80 milligrams of Rezdiffra, and 296 received 100 milligrams of Rezdiffra. Each subject also received “standard care for NASH, which includes counseling for healthy diet and exercise.”
At the 12 month point in the study, “liver biopsies showed that a greater proportion of subjects who were treated with Rezdiffra achieved NASH resolution or an improvement in liver scarring as compared with those who received the placebo.” 26 to 27 percent of those who received 80 milligrams of Rezdiffra and 24 to 36 percent of those who received 100 milligrams “experienced NASH resolution and no worsening of liver scarring, compared to 9 to 13 percent of those who received placebo.” Plus, 23 percent of subjects who received 80 milligrams and 24 to 28 percent of subjects who received 100 milligrams “experienced an improvement in liver scarring and no worsening of NASH, compared to 13 to 15 percent of those who received placebo.” Typically, the disease progresses slowly, so seeing these results after just one year of treatment is “notable.”
Among warnings for patients taking Rezdiffra include to avoid taking the drug if you have decompensated cirrhosis. Additionally, taking it at the same time as other drugs, “in particular statins for lowering cholesterol, may result in potentially significant drug interactions.”
FDA’s website has the release.
