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The FDA has granted accelerated approval to Madrigal Pharmaceuticals’ Resmetilom (Resdifla) for the treatment of metabolic dysfunction-associated steatohepatitis (MASH) in adult patients, the federal agency announced.1 This decision makes resmetilom the first approved treatment for MASH, an advanced form of nonalcoholic fatty liver disease (NAFLD), previously known as nonalcoholic steatohepatitis (NASH).

FDA Headquarters at White Oak Campus in Silver Spring, Maryland/JHVE Photo – Stock.adobe.com

This approval was based on data from a comprehensive clinical development program consisting of 18 clinical studies (12 Phase 1 studies, 2 Phase 2 studies, and 4 Phase 3 studies). A feature of this program is the pivotal MAESTRO-NASH (NCT03900429) trial, which showed resmetirom to have broad therapeutic efficacy in MASH patients with liver fibrosis, reducing low-density lipoprotein (LDL) cholesterol and other related lipid levels. showed a decrease in suffering from heart disease.

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Trial name: Phase 3 study evaluating the efficacy and safety of MGL-3196 (resmetirom) in patients with NASH and fibrosis

ClinicalTrials.gov identifier: NCT03900429

sponsor: madrigal pharmaceutical

summary: In a double-blind, placebo-controlled, randomized phase 3 trial, 80 mg or 100 mg of MGL-3196 resolved NASH and/or reduced fibrosis in liver biopsy and reduced cirrhosis compared to placebo. and/or to determine whether it prevents progression to progressive liver disease.

“Until now, NASH patients with significant liver scarring have had no drugs that directly address their liver damage,” says Nikolai Nikolov, M.D., acting director of the FDA’s Center for Drug Evaluation and Research, Office of Immunology and Inflammation. said in a release.1 “Today’s approval of Rezdifla provides these patients with a treatment option in addition to diet and exercise for the first time.”

Resmetilom is a liver-directed thyroid hormone receptor (THR)-β agonist oral therapy. It is taken once a day and targets the main root cause of MASH in the liver. Thyroid hormone receptor (THR)-β activation is important for liver function, and hepatic (THR)-β activity is lower in MASH patients, which may exacerbate mitochondrial dysfunction, lipotoxicity, and fibrosis. Research shows that.2

Read more: FDA approves Wegovy to reduce cardiovascular risks of overweight and obesity

MAESTRO-NASH is an ongoing, double-blind, placebo-controlled, randomized, phase 3 clinical trial evaluating resmetirom versus placebo for resolution of NASH and improvement of fibrosis.3 The study included 966 adult patients with biopsy-confirmed MASH and fibrosis stage F1B, F2, or F3. Of these, 322 received 80 mg of resmetirom, 323 received 100 mg, and 321 received a placebo. The two primary endpoints at week 52 were resolution of MASH without worsening of fibrosis and at least one grade reduction in fibrosis.

The study found that 25.9% of patients in the 80 mg group and 29.9% of patients in the 100 mg group achieved MASH resolution without worsening of fibrosis, compared with 9.7% of patients in the placebo group. Ta. At least one grade of fibrosis improvement was achieved in 24.2% of patients in the 80 mg group and 25.9% of patients in the 100 mg group compared with 14.2% in the placebo group.

Changes in LDL cholesterol levels from baseline to week 24 were 0.1% in the placebo group, -13.6% in the 80 mg group, and -16.3% in the 100 mg group. Furthermore, diarrhea and nausea were more common in the resmetirom group, and the incidence of serious adverse events was similar in the three groups.

“Continued approval for this indication may be contingent on validation and demonstration of clinical benefit in ongoing confirmatory trials,” Madrigal said in a release.Four The company plans to launch resmetilom in the U.S. in April and will distribute it through a limited network of specialty pharmacies.

“Today is a day of celebration for NASH patients who have waited years for the first approved treatment,” Wayne Eskridge, co-founder and CEO of the Fatty Liver Foundation, said in a release. Stated.Four “We believe this milestone of approval brings new energy and momentum to the NASH community, accelerating efforts to improve disease education, build care pathways, and expand investment in NASH research.”

Read more: FDA Update Resource Center

References
1. FDA approves initial treatment for patients with liver scarring due to fatty liver disease. news release. F.D.A. March 14, 2024. Accessed March 14, 2024. https://www.fda.gov/news-events/press-payments/fda-approves-first-treatment-patients-liver-scarring-due-fatty-liver-disease
2. Resmetilom Clinical Development Program: Leading NASH Research. news release. Madrigal Pharmaceuticals. Accessed March 14, 2024. https://www.madrigalpharma.com/our-programs/resmetirom/
3. Harrison SA, Bedossa P, Guy CD, et al. Phase 3 randomized controlled trial of resmetirom in NASH with liver fibrosis. N English J Medicine. 2024;390(6):497-509. doi:10.1056/NEJMoa2309000
4. Madrigal Pharmaceuticals announces FDA approval of Rezdiffra™ (resmetilom) for the treatment of patients with non-cirrhotic nonalcoholic steatohepatitis (NASH) with moderate to progressive liver fibrosis. news release. Madrigal Pharmaceuticals. March 14, 2024. Accessed March 14, 2024. https://ir.madrigalpharma.com/news-releases/news-release-details/madrigal-pharmaceuticals-announces-fda-approval-rezdiffratm

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