FDA Awaits Final Decision on Madrigal’s Resmetirom for NASH Treatment with Priority Review and NDA Acceptance Set for March 14, 2024
Madrigal Pharmaceuticals is anticipating the U.S. Food and Drug Administration (FDA) decision on the New Drug Application (NDA) for their once daily, oral, thyroid hormone receptor (THR)-β selective agonist, resmetirom, which is being considered for the treatment of adults with nonalcoholic steatohepatitis (NASH) and liver fibrosis. The FDA has granted resmetirom Priority Review and accepted the NDA, with a Prescription Drug User Fee Act (PDUFA) goal date of March 14, 2024.
Resmetirom's clinical development program includes 18 clinical studies, comprising Phase 1 studies, Phase 2 studies, and Phase 3 studies (MAESTRO-NASH, MAESTRO-NAFLD-1, MAESTRO-NAFLD-OLE, and MAESTRO-NASH-OUTCOMES) that demonstrate the potential to target key underlying causes of NASH in the liver. The pivotal Phase 3 MAESTRO-NASH study achieved liver histological improvement endpoints, supporting the potential for accelerated approval. Resmetirom is in an ongoing open-label active treatment extension study of MAESTRO-NAFLD-OLE.
In NASH, thyroid hormone beta activity in the liver is impaired, leading to impaired mitochondrial function and inflammation. Resmetirom aims to treat the liver fibrosis associated with NASH, potentially resolving steatohepatitis that drives negative liver outcomes.
Madrigal's submissions also include publications in the New England Journal of Medicine, providing detailed analyses reinforcing resmetirom's safety profile and the potential to become the first approved medicine for NASH.
Key Details:
- Resmetirom is a THR-β selective agonist, designed to address key underlying causes of NASH
- FDA has granted Priority Review and accepted the NDA
- PDUFA goal date is set for March 14, 2024
- Resmetirom is analyzed based on positive results from the Phase 3 MAESTRO-NASH and additional studies
- The application is for liver fibrosis treatment in adults with NASH
- Resmetirom's potential approval implies the possibility of being the first medicine approved for NASH, addressing a significant unmet needs for NASH treatment and combating the disease's associated leading cause of liver-related mortality