Madrigal's Resmetirom Shows Promise in NASH Treatment Where Others Have Failed
• Madrigal Pharmaceuticals' resmetirom demonstrated significant efficacy in the Phase 3 MAESTRO-NASH trial, achieving both primary endpoints of NASH resolution and liver fibrosis improvement.
• The once-daily oral thyroid hormone receptor β-selective agonist showed NASH resolution in 26-30% of treated patients versus 10% on placebo, positioning it as a potential first-approved therapy for this growing health concern.
• Resmetirom's success stands in stark contrast to Gilead's selonsertib, which failed its Phase 3 STELLAR-4 trial, highlighting the difficulty in developing effective NASH treatments despite the condition's multi-billion dollar market potential.
Maralixibat Demonstrates Efficacy in Treating Pruritus and Reducing Bile Acids in PFIC Patients
• A Phase 3 trial (MARCH-PFIC) reveals that maralixibat significantly improves pruritus in patients with progressive familial intrahepatic cholestasis (PFIC).
• The study showed a significant reduction in serum bile acid concentrations in PFIC patients treated with maralixibat compared to placebo.
• Maralixibat represents a potential non-surgical treatment option for managing PFIC by interrupting enterohepatic circulation.
• The most common adverse event associated with maralixibat was mild to moderate, transient diarrhea.
FDA Approves Rezdiffra (Resmetirom) as First Treatment for NASH with Liver Fibrosis
• The FDA has granted accelerated approval to Rezdiffra (resmetirom) for adults with noncirrhotic NASH and moderate to advanced liver fibrosis.
• Rezdiffra, a THR-β agonist, is the first FDA-approved medication for NASH, also known as MASH, marking a significant milestone.
• Approval was based on Phase 3 MAESTRO-NASH trial data, which demonstrated improved liver fibrosis and NASH resolution compared to placebo.
• Madrigal plans to launch Rezdiffra in the U.S. in April, with a patient support program to aid access and affordability.
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