New FDA Approval: REZDIFFRA™ (resmetirom) Summary
The Food and Drug Administration Recently Approved REZDIFFRA™ (resmetirom) for the treatment of adults with noncirrhotic non-alcoholic steatohepatitis (NASH) with moderate to advanced liver scarring (fibrosis), to be used along with diet and exercise.
Read the InpharmD summary below:
| Drug Name |
REZDIFFRA™ (resmetirom) |
| Active Ingredient |
resmetirom |
| Date of Approval |
3/14/2024 |
| Manufacturer |
Madrigal Pharmaceuticals Inc. |
| Approval Pathways |
NDA; Accelerated Approval |
| Therapeutic Class |
Thyroid hormone receptor-beta (THR-beta) agonist |
| Formulation |
Tablets |
| MoA |
Resmetirom is a partial agonist of the thyroid hormone receptor-beta (THR-β). Resmetirom produced 83.8% of the maximum response compared to triiodothyronine (T3), with an EC50 of 0.21 μM in an in vitro functional assay for THR-β activation. The same functional assay for thyroid hormone receptor- alpha (THR-α) agonism showed 48.6% efficacy for resmetirom relative to T3, with an EC50 of 3.74 μM. THR-β is the major form of THR in the liver, and stimulation of THR-β in the liver reduces intrahepatic triglycerides, whereas actions of thyroid hormone outside the liver, including in heart and bone, are largely mediated through THR-α. |
| Dosing and Administration |
60 mg, 80 mg, and 100 mg
The recommended dosage of REZDIFFRA™ is based on actual body weight.
For patients weighing: <100 kg, the recommended dosage is 80 mg orally once daily. ≥100 kg, the recommended dosage is 100 mg orally once daily. Administer REZDIFFRA with or without food.
|
| Place in Therapy |
REZDIFFRA™ FDA approval is a milestone as the first medication for moderate to advanced liver disease. In trials, over 80% of REZDIFFRA™-treated participants halted or reversed liver scarring.
FDA approval was based on analysis of around 900 MAESTRO-NASH trial participants with Metabolic Dysfunction-Associated Steatohepatitis (MASH) and liver fibrosis, where over 25% experienced MASH resolution without fibrosis worsening, compared to 10% on placebo. The MAESTRO-NAFLD-1 trial showed REZDIFFRA™ safety and tolerability over one year. GLP-1 receptor agonists can improve mild MASH symptoms through weight loss but don't target inflammation or scarring. REZDIFFRA™ directly reduces liver fat buildup, effectively treating MASH and liver scarring.
|
| Expected Market Launch Date |
April 2024 |
| New Molecular Entity (NME) or Existing Formulation |
NME |
| Expected Cost |
Unavailable |
| Product Discontinuation |
N/A |
Share