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      New drug approved for MASH

      Rezdiffra (resmetirom) available for patients with metabolic dysfunction-associated steatohepatitis

       

      Arpana Mathur, MD, MBA, Executive Medical Director of Medical Affairs

      Briefing

      Metabolic dysfunction-associated steatohepatitis (MASH, formerly known as non-alcoholic steatohepatitis, or NASH)1, is a condition, not related to alcohol consumption, in which fat builds up in the liver. It can lead to fibrosis or even cirrhosis, which is irreversible. MASH is most commonly seen in people who are overweight or obese, and represents the fastest growing indication for liver transplantation in Western countries.2

      New treatment approved

      The FDA recently approved an oral medication called resmetirom, sold under the brand name Rezdiffra™ (Madrigal Pharmaceuticals), for adults with MASH who have moderate to advanced scarring of the liver. Approximately 6 to 8 million Americans have MASH, and that number is expected to increase.3 While lifestyle interventions of diet and exercise can help reverse fatty liver disease, it may not reverse the fibrosis caused by MASH. The drug works to reduce liver fat accumulation and has been shown to improve scarring in about 25 percent of people who take it.4 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.

       

      Diagnosis and drug availability

      Though clinical trial participants did have to undergo a biopsy to be part of the study, prescribing notes do not include a liver biopsy requirement for diagnosis.5 Rezdiffra is distributed through a limited specialty pharmacy network and has an annual wholesale price of $47,400.6

       

      Side effects

      The most common side effects of Rezdiffra include diarrhea and nausea; warnings include drug-induced liver toxicity and gallbladder-related side effects. A separate study evaluated the safety and tolerability of the drug and contributed to the safety database supporting benefit-risk assessment.7

       

      Other drugs in the MASH pipeline

      There are other drugs in the pipeline aimed at decreasing the amount of liver fibrosis:

      • Ozempic (semaglutide, Novo Nordisk) – Developed to treat diabetes, this long-acting glucagon like peptide-1 (GLP-1) analogue injectable is projected to be approved in 2025.8
      • Lanifibranor (Inventiva Pharma) – This oral medication acts to induce anti-fibrotic, anti-inflammatory as well as beneficial metabolic changes and is projected to be approved in 2027.9
      • Lipaglyn (saroglitazar, Zydus) – This oral medication combines lipid and glucose lowering effects into a single molecule and is projected to be approved in 2027.10
      • Efruxifermin (Akero Therapeutics/ Amgen) – Engineered to mimic the biological activity of FGF21, which regulates multiple metabolic pathways and cellular processes, this injectable is projected to be approved in 2028.11

       

      Related tags

      Discover additional insights

      • 1 Nonalcoholic Steatohepatitis (NASH) https://liverfoundation.org/liver-diseases/fatty-liver-disease/nonalcoholic-steatohepatitis-nash/

      • 2 NAFLD and liver transplantation: Disease burden, current management and future challenges https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7607500/

      • 3 FDA Approves First Treatment for Patients with Liver Scarring Due to Fatty Liver Disease https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-liver-scarring-due-fatty-liver-disease

      • 4 Ibid.

      • 5 Madrigal Pharmaceuticals Announces FDA Approval of Rezdiffra™ (resmetirom) for the Treatment of Patients with Noncirrhotic Nonalcoholic Steatohepatitis (NASH) with Moderate to Advanced Liver Fibrosis https://ir.madrigalpharma.com/news-releases/news-release-details/madrigal-pharmaceuticals-announces-fda-approval-rezdiffratm

      • 6 US FDA approves first drug for fatty liver disease NASH https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-approves-first-drug-fatty-liver-disease-nash-2024-03-14/

      • 7 https://ir.madrigalpharma.com/news-releases/news-release-details/madrigal-pharmaceuticals-announces-fda-approval-rezdiffratm

      • 8 https://www.novonordisk.com/science-and-technology/r-d-pipeline.html

      • 9 https://inventivapharma.com/pipeline/lanifibranor/

      • 10 https://www.lipaglyn.com/about_lipaglyn.html

      • 11 https://akerotx.com/efruxifermin/

       

      This document contains references to brand-name prescription drugs that are trademarks or registered trademarks of pharmaceutical manufacturers not affiliated with CVS Health.