Madrigal Announces US Availability of Resmetirom (Rezdiffra), First Patients Treated for NASH

Resmetirom to treat NASH with F2-F3 fibrosis, the only approved therapy, is available through Madrigal's specialty pharmacy network, the company said.

Resmetirom (Rezdiffra) is now available for the treatment of patients with noncirrhotic nonalcoholic steatohepatitis (NASH) through Madrigal Pharmaceuticals’ specialty pharmacy network, the company announced today.1 The first eligible patients have received the novel oral thyroid hormone receptor (THR)-β selective agonist, the only drug currently approved by the US Food and Drug Administration for the treatment of noncirrhotic NASH with moderate to advanced (stage F2 to F3) hepatic fibrosis.

“The introduction of Rezdiffra is completely changing the conversation about NASH in our practice. I can finally tell my appropriate patients with moderate to advanced fibrosis that we have an approved treatment to help improve fibrosis and resolve NASH. Our team at Arizona Liver Health has never felt more energized, and we recently celebrated when our first patient received Rezdiffra," Naim Alkhouri, MD, chief medical officer, chief of transplant hepatology, and director of the Fatty Liver Program at Arizona Liver Health said in the news release.1

The accelerated approval of resmetirom in March 2024 was based on findings from the pivotal phase 3 MAESTRO-NASH trial, which continues as an outcomes study to generate confirmatory data, according to Madrigal. If positive, those data will help verify the agent’s clinical benefit and support its full approval. In a separate ongoing outcomes trial, Madrigal is also evaluating the impact of resmetirom vs placebo on progression to hepatic decompensation in individuals with well-compensated NASH cirrhosis.

“Years of advocacy from the NASH community helped pave the way for Rezdiffra as the first approved medication for this long-neglected disease,” Donna R Cryer, JD, founder and chief executive officer of the Global Liver Institute, in the press statement.1

“We were thrilled to see that the Rezdiffra prescribing information is patient-centric: there is no biopsy requirement for diagnosis. Moving the field away from biopsy has been a long-time goal for the Global Liver Institute and I believe the availability of Rezdiffra will help grow patient awareness of noninvasive testing options for NASH.”1

The resmetirom indication calls for the drug to be administered in conjunction with dietary changes and enhanced physical activity. The labeled dosage of resmetirom is based on actual body weight: 80 mg orally once daily for patients weighing less than 100 kg (220 lbs), and 100 mg orally once daily for patients weighing 100 kg or more.

The most common adverse reactions reported while taking resmetirom are diarrhea and nausea, which begin early in treatment and are typically mild to moderate in severity, and pruritis, vomiting, constipation, abdominal pain, and dizziness.

Madrigal has established programs to help appropriate patients who may benefit from resmetirom access treatment, including those with no insurance or who may be facing affordability challenges.

Source: Madrigal Pharmaceuticals Announces U.S. Availability of Rezdiffra (resmetirom) for the Treatment of Patients with Noncirrhotic NASH with Moderate to Advanced Liver Fibrosis. News release. Madrigal Pharmaceuticals. April 9, 2024. Accessed April 9, 2024. https://ir.madrigalpharma.com/news-releases/news-release-details/madrigal-pharmaceuticals-announces-us-availability-rezdiffratm