Today, the U.S. Food and Drug Administration approved Rezdiffra (resmetirom) for the treatment of adults with noncirrhotic non-alcoholic steatohepatitis (NASH) with moderate to advanced liver scarring (fibrosis), to be used along with diet and exercise,
“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 Centre 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.”
NASH happens when the liver gets inflamed and scarred because of non-alcoholic fatty liver disease. It's often linked with health issues like high blood pressure and type 2 diabetes. Around 6-8 million people in the U.S. might have NASH with serious liver scarring, and this number could go up.
Rezdiffra helps by partially activating a receptor in the liver, reducing fat buildup. Its safety and effectiveness were checked in a 54-month study where its effect on liver inflammation and scarring was measured. Now, the makers need to do another study after approval to confirm Rezdiffra's benefits. This will be the same 54-month study, which is still ongoing.
To join the study, patients needed a liver biopsy showing NASH-related inflammation with moderate or advanced scarring. In the study, 888 people were randomly given either a placebo, 80 milligrams of Rezdiffra, or 100 milligrams of Rezdiffra once a day, along with regular NASH care like diet and exercise counselling.
Rezdiffra's common side effects are diarrhea and nausea. It's important to watch out for liver toxicity and gallbladder issues. Avoid Rezdiffra in advanced cirrhosis. Stop treatment if liver function worsens. Using Rezdiffra with statins can lead to significant interactions. Healthcare providers should check the full prescribing information for dosage adjustments and potential drug interactions.
The FDA approved Rezdiffra under the accelerated approval pathway, which allows for earlier approval of drugs that treat serious conditions and address an unmet medical need, based on a surrogate or intermediate clinical endpoint that is reasonably likely to predict clinical benefit. The required aforementioned 54-month study, which is ongoing, will assess clinical benefit after 54 months of Rezdiffra treatment, Rezdiffra received Breakthrough Therapy, Fast Track and Priority Review designations for this indication, The FDA granted the approval of Rezdiffra to Madrigal Pharmaceuticals.
Learn more at: FDA Approves First Treatment for Patients with Liver Scarring Due to Fatty Liver Disease | FDA
