CONSHOHOCKEN, Pa. - Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL) announced today that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Rezdiffra (resmetirom), a new medication for the treatment of nonalcoholic steatohepatitis (NASH) with moderate to advanced liver fibrosis in adults.
This marks the first FDA-approved therapy for the condition, which until now had no approved treatment options.
Rezdiffra, which is to be used in conjunction with diet and exercise, received approval based on Phase 3 trial results, demonstrating improved liver fibrosis and resolution of NASH in patients with noncirrhotic NASH with moderate to advanced liver fibrosis. Notably, the prescribing information for Rezdiffra does not require a liver biopsy for diagnosis.
The Phase 3 MAESTRO-NASH trial, which enrolled 1,759 patients with biopsy-confirmed NASH, showed that Rezdiffra, at doses of 80 mg and 100 mg, significantly improved NASH resolution and fibrosis compared to placebo after 52 weeks of treatment. The study is ongoing as an outcomes trial to confirm the clinical benefit of Rezdiffra, which could lead to full approval.
Madrigal's CEO, Bill Sibold, expressed that the approval is a historic moment for the NASH field, resulting from over 15 years of research. The company aims to change the treatment paradigm for NASH, offering a liver-directed therapy to improve fibrosis and resolve NASH before the progression to cirrhosis.
Wayne Eskridge, CEO of the Fatty Liver Foundation, also celebrated the approval, highlighting the potential to bring new energy to the NASH community.
Rezdiffra is expected to be available in the U.S. in April and will be distributed through a specialty pharmacy network. Madrigal has established a patient support program to assist with insurance and affordability challenges, including co-pay support for eligible patients, and a patient assistance program for those without insurance.
The most common adverse reactions reported in patients treated with Rezdiffra included diarrhea, nausea, pruritis, abdominal pain, vomiting, constipation, and dizziness, with diarrhea and nausea typically being mild to moderate in severity.
Madrigal will host a conference call today at 5:15 PM ET to discuss the accelerated approval of Rezdiffra. This article is based on a press release statement from Madrigal Pharmaceuticals.
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