FDA Grants Breakthrough Therapy Designation to Madrigal’s Resmetirom

On April 18, 2023, Madrigal Pharmaceuticals, a clinical-stage biopharmaceutical company, announced that its drug candidate resmetirom has received the Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of non-alcoholic steatohepatitis (NASH). This designation is given to drugs that have shown significant improvement in treating serious or life-threatening conditions and have the potential to address unmet medical needs.

Nonalcoholic steatohepatitis (NASH) is a more severe form of nonalcoholic fatty liver disease (NAFLD). NAFLD is thought to affect more than 20% of adults worldwide and approximately 30% in the United States. 20% of that population may have NASH. NASH is a leading cause of liver-related mortality and a growing burden on healthcare systems worldwide.

Furthermore, patients with NASH, particularly those with more advanced metabolic risk factors (hypertension, concomitant type 2 diabetes), are at increased risk for adverse cardiovascular events, morbidity, and mortality. Currently, there are no FDA-approved treatments for NASH, and resmetirom has shown promising results in clinical trials.

In addition to the Breakthrough Therapy designation, Madrigal Pharmaceuticals also announced the completion of enrollment in the Phase 3 MAESTRO-NASH biopsy trial for resmetirom. This trial is evaluating the safety and efficacy of resmetirom in NASH patients, and the results are expected to support the regulatory submission for approval. 

About Madrigal Pharmaceuticals

Madrigal Pharmaceuticals, Inc. (Nasdaq: MDGL) is a clinical-stage biopharmaceutical company focused on developing novel therapies for nonalcoholic steatohepatitis (NASH), a liver disease with significant unmet medical needs. Resmetirom, Madrigal’s lead candidate, is a once-daily, oral, THR-selective agonist designed to target key underlying causes of NASH in the liver. 

For more information, please visit: Breakthrough Therapy Designation to Madrigal’s Resmetirom

FDA News and Insights – Recommended Articles 

• FDA Approves Medtronic MiniMed 780G System
• FDA Grants Fast Track Designation to Botensilimab Plus Balstilimab
• FDA Orphan Drug Designation for XORTX ADPKD Treatment
• Orphan Drug Designation: Key Developments in August 2023
• Orphan Drug Designation: Updates and Highlights for September 2023
• Lenire: Innovative Treatment for Tinnitus Relief
• LuxCreo Launches LuxAlign: 3D Printed Clear Aligners Revolutionizing Dental Care