The landscape of liver disease treatment underwent transformative changes in 2024, marked by groundbreaking regulatory approvals and significant clinical advances across multiple conditions. These developments promise new hope for millions of patients worldwide struggling with various forms of liver disease.
Madrigal Pharmaceuticals achieved a historic milestone with Rezdiffra (resmetirom), securing the first-ever FDA approval for treating metabolic dysfunction-associated steatohepatitis (MASH). The approval was supported by compelling Phase 3 MAESTRO-NASH trial results, which demonstrated significant improvements in both disease resolution and liver scarring reduction after one year of daily treatment.
The obesity and diabetes medication front showed unexpected promise in liver disease treatment. Eli Lilly's tirzepatide demonstrated remarkable efficacy in MASH treatment during Phase 2 trials, with dose-dependent improvements in disease resolution and liver fibrosis reduction, suggesting potential therapeutic repurposing opportunities.
Advances in Rare Liver Diseases
Primary sclerosing cholangitis (PSC) research saw notable progress, with multiple therapeutic candidates advancing through clinical development. LISCure Biosciences' probiotic therapy LB-P8 received FDA fast track designation, while Chemomab Therapeutics secured important patents in Brazil and Israel for CM-101, their experimental PSC treatment.
Hepatitis B Treatment Innovation
The viral hepatitis landscape witnessed significant developments, particularly in chronic hepatitis B treatment. GSK's bepirovirsen received FDA fast track designation following impressive Phase 2b results, where patients achieved sustained viral suppression. Additionally, Chroma Medicine's experimental one-time therapy, CRMA-1001, showed promising results in preclinical studies, nearly eliminating key viral markers in animal models.
Research Infrastructure Development
The U.S. government announced its first national study on metabolic dysfunction-associated steatotic liver disease (MASLD), signaling increased attention to this growing public health concern. This comprehensive study aims to assess disease prevalence, diagnostic and treatment costs, and the economic impact of disease progression.
Clinical Trial Momentum
Akero Therapeutics launched an ambitious clinical program with three Phase 3 trials of efruxifermin for fatty liver disease, collectively aiming to enroll over 2,800 patients globally. The SYNCHRONY program's comprehensive approach includes studies in both compensated cirrhosis and non-cirrhotic MASH patients, as well as real-world effectiveness evaluation.
The rapid pace of development in liver disease therapeutics reflects growing recognition of the urgent need for effective treatments. With multiple promising candidates in late-stage development and increased regulatory support through fast-track designations, the field appears poised for continued advancement in the coming years.
