Resmetirom
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| Trade names | Rezdiffra |
| Other names | MGL-3196 |
| AHFS/Drugs.com | Monograph |
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| Routes of administration | By mouth |
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| Formula | C17H12Cl2N6O4 |
| Molar mass | 435.22 g·mol−1 |
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Resmetirom, sold under the brand name Rezdiffra, is a medication used for the treatment of noncirrhotic nonalcoholic steatohepatitis.[1] It is a thyroid hormone receptor beta (NR1A2) agonist.[1]
The most common side effects include diarrhea and nausea.[2]
Resmetirom was approved for medical use in the United States in March 2024.[2][3][4]
Medical uses
[edit]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
[edit]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]
References
[edit]- ^ a b c d "Rezdiffra- resmetirom tablet, coated; Rezdiffra- resmetirom tablet, coated; Rezdiffra- resmetirom tablet, coated". DailyMed. 14 March 2024. Archived from the original on 2 April 2024. Retrieved 2 April 2024.
- ^ a b c d e f g h i "FDA Approves First Treatment for Patients with Liver Scarring Due to Fatty Liver Disease". U.S. Food and Drug Administration (FDA) (Press release). 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. - ^ "Novel Drug Approvals for 2024". U.S. Food and Drug Administration (FDA). 29 April 2024. Archived from the original on 30 April 2024. Retrieved 30 April 2024.
- ^ "Madrigal Pharmaceuticals Announces FDA Approval of Rezdiffra (resmetirom) for the Treatment of Patients with Noncirrhotic Nonalcoholic Steatohepatitis (NASH) with Moderate to Advanced Liver Fibrosis". Madrigal Pharmaceuticals (Press release). 14 March 2024. Archived from the original on 14 March 2024. Retrieved 14 March 2024.
- ^ Harrison SA, Bedossa P, Guy CD, Schattenberg JM, Loomba R, Taub R, et al. (February 2024). "A Phase 3, Randomized, Controlled Trial of Resmetirom in NASH with Liver Fibrosis". The New England Journal of Medicine. 390 (6): 497–509. doi:10.1056/NEJMoa2309000. hdl:11573/1706995. PMID 38324483. S2CID 267544939.
