Naim Alkhouri, MD: First Real-World Data for Resmetirom Use in Clinical Practice

Findings from a recent study provide the first real-world data on the use of resmetirom (Rezdiffra) in patients with metabolic dysfunction-associated steatohepatitis (MASH) in clinical practice.¹

In the study, data were collected from 72 patients with MASH who were prescribed resmetirom at 2 US tertiary care centers between March and May 2024 to describe the clinical characteristics and noninvasive test (NIT) results of real-world patients versus those enrolled in phase 3 resmetirom clinical trials.¹

On March 14, 2024, resmetirom became the first and only US Food and Drug Administration-approved MASH therapeutic, earning accelerated approval for noncirrhotic MASH with moderate to advanced fibrosis based on 18 clinical studies in the oral, thyroid hormone receptor-β selective agonist’s clinical development program.²

“This has been a historic approval for us and the MASLD and MASH community as the first and only FDA-approved medication that we have been using in our clinics now for the past 6 months or so with great success,” Naim Alkhouri, MD, chief medical officer, chief of transplant hepatology, and director of the Fatty Liver Program at Arizona Liver Health, said to HCPLive.

At the American College of Gastroenterology (ACG) 2024 Annual Scientific Meeting in Philadelphia, Pennsylvania, Alkhouri presented data on the first 72 patients with MASH who were prescribed resmetirom at Arizona Liver Health and Indiana University following the agent’s FDA approval.¹

Among the cohort, the median age was 56 years (range, 20–80), with males comprising 40.3% of the cohort. Resmetirom was prescribed at doses of 60 mg (1.4%), 80 mg (47.2%), and 100 mg (51.4%). The prevalence of obesity and type 2 diabetes was 79% and 41.7%, respectively.¹

Concomitant medications included insulin (5.6%), GLP-1 agonists (23.6%), statins (41.7%), and aspirin (20.8%). Fibroscan data revealed 45.8% of patients had a liver stiffness measurement between 10–20 kPa, and 80.6% had a CAP ≥ 280 dB/m, indicating advanced fibrosis and steatosis, respectively.¹

“What we are showing here is that in the real world, we are able to identify appropriate patients to be prescribed resmetirom based on noninvasive testing without the need for liver biopsy, and that the patient characteristics and the NIT data are very consistent with MAESTRO-NASH, implicating that we can really replicate the results from the phase 3 program, hopefully, in the real world,” Alkhouri said. “This is the first glimpse. We continue to collect more data… Our goal is to demonstrate that resmetirom was equally as effective in the real world as in the phase 3 clinical trials. More to come on this, but we are very excited to share the first real-world data on the use of resmetirom in the United States.”

Editors’ note: Alkhouri has relevant disclosures with Altimmune, Chronwell, Cytodyne, Madrigal Pharmaceuticals, Merck, Novo Nordisk, Rivus, Takeda, and Terns.

References

1. ^{Kaya E, Nadeem R, Kapoor H, et al. P4645 - Real-World Characteristics of the Patients With Metabolic Dysfunction-Associated Steatohepatitis Prescribed Resmetirom: Data Derived From Two Tertiary Care Centers in the United States. Paper presented at: ACG 2024 Annual Scientific Meeting. Philadelphia, Pennsylvania. October 25-30, 2024.}
2. ^{Brooks, A. Resmetirom (Rezdiffra) Receives Historic FDA Approval for Noncirrhotic NASH. HCPLive. March 14, 2024. Accessed November 11, 2024. https://www.hcplive.com/view/resmetirom-rezdiffra-receives-historic-fda-approval-for-noncirrhotic-nash}