Chemistry:Resmetirom
Resmetirom, sold under the brand name Rezdiffra, is a medication used to treat a form of fatty liver disease. Specifically it is used in the treatment of noncirrhotic metabolic dysfunction associated steatohepatitis (MASH) (formerly known as nonalcoholic steatohepatitis NASH).[1] It is structurally similar to the thyroid hormone triiodothyronine and acts as an agonist of the thyroid hormone receptor beta (NR1A2).[1]
The most common side effects include diarrhea and nausea.[2]
Resmetirom was approved for medical use in the US in March 2024,[2] and in the European Union in August 2025.[3] The US Food and Drug Administration considers it to be a first-in-class medication.[4]
Medical uses
Resmetirom is indicated, in conjunction with diet and exercise, for the treatment of adults with noncirrhotic nonalcoholic steatohepatitis with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis).[1][2]
History
In a phase III clinical trial, it was found to be effective for resolving noncirrhotic nonalcoholic steatohepatitis and improving liver fibrosis.[5]
The US Food and Drug Administration (FDA) evaluated the safety and efficacy of resmetirom based on an analysis of a surrogate endpoint at month twelve in a 54-month, randomized, double-blind placebo-controlled trial.[2] The surrogate endpoint measured the extent of liver inflammation and scarring.[2] The FDA requires the sponsor to conduct a postapproval study to verify and describe the clinical benefit of resmetirom, which will be done through completing the same 54-month study.[2] In the trial, 888 participants were randomly assigned to receive one of the following: placebo (294 participants); 80 milligrams of resmetirom (298 participants); or 100 milligrams of resmetirom (296 participants); once daily, in addition to standard care for noncirrhotic nonalcoholic steatohepatitis, which includes counseling for healthy diet and exercise.[2]
The FDA granted the application for resmetirom accelerated approval, along with breakthrough therapy, fast track, and priority review designations.[2] The FDA granted the approval of Rezdiffra to Madrigal Pharmaceuticals.[2]
Society and culture
Legal status
Resmetirom was approved for medical use in the US in March 2024.[6]
In June 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a conditional marketing authorization for the medicinal product Rezdiffra, intended for the treatment of adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH).[7] The applicant for this medicinal product is Madrigal Pharmaceuticals EU Limited.[7][8] Resmetirom was authorized for medical use in the European Union in August 2025.[7][3]
Research
A systematic review and meta-analysis of resmetirom, published in 2024, found that it is well tolerated and that it improves hepatic fat content, liver enzymes, and fibrosis biomarkers in people with non-alcoholic steatohepatitis (NASH).[9]
References
- ↑ 1.0 1.1 1.2 Cite error: Invalid
<ref>tag; no text was provided for refs namedRezdiffra FDA label - ↑ 2.0 2.1 2.2 2.3 2.4 2.5 2.6 2.7 2.8 "FDA Approves First Treatment for Patients with Liver Scarring Due to Fatty Liver Disease". FDA.gov (Press release). US: Food and Drug Administration. 14 March 2024. Archived from the original on 14 March 2024. Retrieved 14 March 2024.
This article incorporates text from this source, which is in the public domain.
- ↑ 3.0 3.1 "Rezdiffra PI". 19 August 2025. https://ec.europa.eu/health/documents/community-register/html/h1962.htm.
- ↑ (PDF) New Drug Therapy Approvals 2024 (Report). US: Food and Drug Administration. January 2025. https://www.fda.gov/media/184967/download. Retrieved 21 January 2025.
- ↑ "A Phase 3, Randomized, Controlled Trial of Resmetirom in NASH with Liver Fibrosis". The New England Journal of Medicine 390 (6): 497–509. February 2024. doi:10.1056/NEJMoa2309000. PMID 38324483.
- ↑ "Madrigal Pharmaceuticals Announces FDA Approval of Rezdiffra (resmetirom) for the Treatment of Patients with Noncirrhotic Nonalcoholic Steatohepatitis (NASH) with Moderate to Advanced Liver Fibrosis". madrigalpharma.com (Press release). Madrigal Pharmaceuticals. 14 March 2024. Archived from the original on 14 March 2024. Retrieved 14 March 2024.
- ↑ 7.0 7.1 7.2 "Rezdiffra EPAR". European Medicines Agency. 20 June 2025. https://www.ema.europa.eu/en/medicines/human/EPAR/rezdiffra. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ "First treatment against liver scarring caused by a type of 'fatty liver disease'". ema.europa.eu (Press release). European Medicines Agency. 20 June 2025. Retrieved 26 June 2025.
- ↑ "Role of Resmetirom, a Liver-Directed, Thyroid Hormone Receptor Beta-Selective Agonist, in Managing Nonalcoholic Steatohepatitis: A Systematic Review and Meta-Analysis". Endocrine Practice 30 (7): 631–638. July 2024. doi:10.1016/j.eprac.2024.04.016. PMID 38697306.
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