FDA Approves First Drug Targeting Liver Scarring from MASH in US

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Clinical trials demonstrated Rezdiffra's ability to resolve NASH or improve liver scarring in a significant proportion of patients.

 

US Liver Cirrhosis Treatment News

The US liver cirrhosis treatment landscape is evolving with a focus on managing the complications of the disease and addressing the underlying causes. While a cure for advanced cirrhosis remains elusive without liver transplantation, recent news highlights promising developments in slowing progression and improving patient outcomes.

Focus on Addressing Underlying Causes:

A growing emphasis is being placed on treating the root causes of liver cirrhosis. For alcohol-associated liver disease, multidisciplinary clinics like the HALT program at UCSF are integrating addiction care with hepatology, showing significant improvements in patient remission rates and liver disease severity markers. For non-alcoholic steatohepatitis (NASH), now known as metabolic dysfunction-associated steatohepatitis (MASH), the FDA approved Rezdiffra (resmetirom) in March 2024. This is the first medication specifically targeting liver scarring due to MASH and is to be used alongside diet and exercise. Clinical trials demonstrated Rezdiffra's ability to resolve NASH or improve liver scarring in a significant proportion of patients.

Emerging Therapies and Clinical Trials:

Several companies are actively involved in developing new treatments for liver cirrhosis and its complications. Namodenoson, an orally bioavailable drug, showed promising results in a clinical case study, leading to the disappearance of decompensated liver cirrhosis episodes. The US market for liver cirrhosis treatment is projected to reach $15 billion by 2030, indicating the significant unmet need and potential for new therapies.

Cell Therapy Shows Promise:

A novel cell therapy using macrophage immune cells derived from the patient's own cells demonstrated significant progress in a clinical trial for advanced liver cirrhosis. The treatment reduced serious liver-related complications and potentially delayed the need for liver transplantation in the study group.

Managing Complications Remains Crucial:

Managing the complications of cirrhosis, such as fluid buildup (ascites), variceal bleeding, and hepatic encephalopathy, remains a cornerstone of treatment. Advances in interventional radiology techniques and the approval of terlipressin for hepatorenal syndrome have improved the management of these severe complications. Nutritional support and addressing malnutrition, frailty, and sarcopenia are also increasingly recognized as important aspects of care.

Future Directions:

Research continues to explore direct antifibrotic approaches, targeting specific cell types and signaling pathways involved in liver scarring. Combination therapies, including systemic agents like semaglutide, are also being evaluated. The development of reliable non-invasive markers for assessing liver fibrosis is crucial for identifying patients who would benefit most from emerging treatments.

Overall:

The US liver cirrhosis treatment landscape is evolving with a multi-pronged approach. While liver transplantation remains the only curative option for end-stage disease, new medications targeting specific causes like MASH, promising cell therapies, and improved management of complications offer hope for slowing disease progression and improving the quality of life for individuals living with liver cirrhosis. Multidisciplinary care models that address underlying conditions like alcohol use disorder are also gaining traction.

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