The FDA Approval of Resmetirom for MASH Changes Everything—for Patients and their Physicians

"The fact that we have a new treatment now for our patients, this is a new hope and it's really changing everything we do."

Naim Alkhouri, MD, chief medical officer, chief of transplant hepatology, and director of the fatty liver program at Arizona Liver Health, in Phoenix, a veteran of the new drug development process, and a member of the research team that saw resmetirom through to the March 2024 FDA approval, talks with Patient Care here about the "watershed moment" for investigators and then switches gears to focus on what the historic approval means for patients. (Follow Dr Alkhouri on X @AlkhouriNaim)

Resmetirom is an oral, thyroid hormone receptor (THR)-β selective agonist that increases hepatic fat metabolism and reduces lipotoxicity and made medical and pharmaceutical history as the first-ever drug approved to treat individuals with metabolic dysfunction associated steatohepatitis with moderate to severe fibrosis. The drug's accelerated FDA approval was based on Madrigal Pharmaceutical's most recent phase 3 data from the MAESTRO-NASH trial, 1 of 4 phase 3 studies in the resmetirom clinical development program. MAESTRO-NASH continues as an outcomes study designed to generate confirmatory data that may be used to verify the drug’s clinical benefit and to support full approval. MAESTRO-NASH is 1 of 18 studies in the Madrigal clinical development program that supported the original 2023 NDA.

Other videos in this series with Dr Alkhouri:

• A MASLD Mantra to Practice By: Screen, Stage, Treat

• The Pipeline for New MASLD & MASH Treatments is Promising, says Naim Alkhouri, MD

• Early Detection of MASLD in Primary Care: The Essentials with Naim Alkhouri, MD

• The Rate of Undetected MASLD in the US is a Sobering Statistic, says Expert Hepatologist Naim Alkhouri, MD