Rezdiffra FDA Approval Status
Yes, Rezdiffra (resmetirom) is FDA-approved—it is the first FDA-approved drug specifically for the treatment of NAFLD/NASH. 1
Background Context
For over two decades, no pharmacological treatment existed for NASH despite its status as the most common chronic liver disease in the United States, affecting 80-100 million Americans. 2 Multiple guidelines from 2015-2016 consistently stated that no FDA-approved drugs were available for NASH treatment. 2, 3, 4, 5
Clinical Trial Evidence Supporting Approval
Rezdiffra's approval was based on clinical trial NCT03900429, which demonstrated:
- Effective improvement in fibrosis by at least one stage without worsening of NAFLD activity score (NAS) 1
- Improvement in lipid profile of NASH patients using 80 or 100 mg doses of resmetirom 1
- The trial was funded by Madrigal Pharmaceuticals, the manufacturer 1
Mechanism and Clinical Significance
Resmetirom (MGL-3196) is a thyroid hormone receptor beta-selective agonist that targets hepatic lipid metabolism. 1 This approval represents a paradigm shift in NASH management, as previous treatment was limited to:
- Lifestyle modifications (7-10% weight loss target) 2, 6
- Off-label medications like pioglitazone and vitamin E 2
- Management of metabolic comorbidities 7
Important Caveats
While Rezdiffra is now FDA-approved, the evidence base remains limited to the single pivotal trial. 1 Clinicians should note that:
- Lifestyle modification remains the cornerstone of NASH treatment regardless of pharmacotherapy 2, 6
- Cardiovascular disease remains the leading cause of death in NAFLD patients, requiring aggressive management of all metabolic risk factors 7, 6
- The drug specifically targets patients with NASH and fibrosis, not simple steatosis 1