Fatty liver disease is one of the most neglected health issues with severe complications. Fatty liver disease, also known as hepatic steatosis, is characterized by an accumulation of fat in the liver cells. This buildup of fat can lead to liver inflammation and damage over time. Obesity, high cholesterol, type 2 diabetes, and excessive alcohol consumption are commonly associated with it.
Non-alcoholic fatty liver disease (NAFLD) is prevalent among individuals who do not consume excessive alcohol. Fatty liver disease can progress to more severe conditions like non-alcoholic steatohepatitis (NASH), liver cirrhosis, or even liver failure if not taken care of.
The first medication for non-alcoholic steatohepatitis (NASH), a severe form of fatty liver disease, was approved by the US Food and Drug Administration (FDA) on Thursday. NASH affects approximately 6-8 million people in the United States, and is often associated with other health problems such as high blood pressure, type 2 diabetes, obesity and high blood fat levels.
What is NASH?
Non-alcoholic steatohepatitis (NASH) is a more severe form of non-alcoholic fatty liver disease (NAFLD), characterized by liver inflammation and damage, along with fat accumulation in the liver. NASH involves inflammation and can progress to liver fibrosis, cirrhosis, and potentially liver cancer. NAFLD may not cause NASH in some individuals, but others may progress to this advanced stage.
Nonspecific symptoms of NASH can include fatigue, weakness, weight loss, and abdominal discomfort. Diagnosis often involves imaging tests and liver biopsies to assess the extent of liver damage. Treatment for NASH focuses on lifestyle modifications such as weight loss, dietary changes, exercise, and managing underlying conditions such as diabetes and high cholesterol. Sometimes liver transplantation and medications may be necessary in more advanced cases.
All about the new medication
Rezdiffra, also known as resmetirom molecule, is an oral drug that targets the underlying causes of NASH. US patients will have access to the drug in April.
Madrigal Pharmaceuticals’ Rezdiffra was shown to improve liver scarring in a clinical trial involving hundreds of people with non-alcoholic steatohepatitis (NASH), the worst form of the condition caused by a buildup of fat in the liver. “Previously, patients with NASH who also have notable liver scarring did not have a medication that could directly address their liver damage,” said the FDA’s Nikolay Nikolov. “Today’s approval of Rezdiffra will, for the first time, provide a treatment option for these patients, in addition to diet and exercise.
Madrigal’s drug wins first US approval for fatty liver disease NASH as rivals circle
Madrigal Pharmaceuticals’ (MDGL.O), drug became the first to win regulatory approval to treat a serious type of fatty liver disease formerly known as non-alcoholic steatohepatitis (NASH) as the race to tap a multi-billion dollar market intensifies.
The approval on Thursday follows limited success in developing a treatment for the disease, which is closely tied to obesity and is spreading quickly.
Treatments for NASH, also known as metabolic dysfunction-associated steatohepatitis, are being developed by the following companies:
| Company | Drug name / developmental stage | Further plans | Recent updates |
| Akero Therapeutics (AKRO.O) | Efruxifermin (Mid-stage) | Drug being tested in two ongoing late-stage studies and a separate mid-stage study | The drug significantly reduced scarring after nearly two years in the mid-stage study, data showed in March |
| Altimmune (ALT.O) | Pemvidutide (Mid-stage) | Mid-stage trial data for the drug expected in first quarter of 2025 | Drug, in December 2022, achieved more than 75% reduction in liver fat content, compared to placebo in an early-stage trial |
| Boehringer Ingelheim and Zealand Pharma (ZELA.CO) | Survodutide (Mid-stage) | Full trial data to be presented in coming months | Trial data from February showed significant improvement in liver inflammation and damage without worsening fibrosis or scarring |
| Eli Lilly (LLY.N) | Tirzepatide (Mid-stage) | – | The drug helped up to 74% of patients achieve “absence of the disease” at 52 weeks, compared to 13% of patients on placebo |
| Ionis Pharmaceuticals Inc (IONS.O) | ION224 (Mid-stage) | – | Trial data published in March showed significant improvement in liver inflammation and damage without worsening fibrosis or scarring |
| Merck & Co (MRK.N) | Efinopegdutide (Mid-stage) | – | Mid-stage trial data from June showed that efinopegdutide significantly reduced liver fat in patients compared to semaglutide |
| Novo Nordisk (NOVOb.CO) | Semaglutide (Late-stage) | Late-stage study expected to enroll about 1,200 patients | No data |
| Viking Therapeutics (VKTX.O) | VK2809 (Mid-stage) | Additional data from the study to be presented in the first half of 2024 | The drug, in May, helped significantly reduce liver fat in patients |
| 89bio (ETNB.O) | Pegozafermin (Late-stage) | Company expects to begin second late-stage trial in MASH patients in the second quarter of 2024 | Began first late-stage study for the drug on Wednesday, which is intended to support a filing for accelerated approval in the U.S. |
