Madrigal Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review its New Drug Application (NDA) for resmetirom for the treatment of adult patients with NASH with liver fibrosis. The FDA has granted Priority Review and assigned a Prescription Drug User Fee Act date for resmetirom of March 14, 2024, the target date by which the FDA intends to complete its review and take action on the NDA. The Agency noted that it is not currently planning to hold an advisory committee meeting to discuss the application.
Resmetirom is a once daily, oral, thyroid hormone receptor (THR)-β selective agonist designed to target key underlying causes of NASH in the liver. The clinical development program for resmetirom is comprised of 18 clinical studies supporting the NDA: twelve Phase 1 studies, two Phase 2 studies, and four Phase 3 studies. Madrigal is seeking approval of resmetirom for the treatment of patients with NASH and liver fibrosis under the FDA’s accelerated approval pathway.
Bill Sibold, Chief Executive Officer of Madrigal, stated, “NASH with liver fibrosis represents a significant unmet need in healthcare today: the disease has a serious impact on patients and, without treatment, it can lead to increased risk of cirrhosis, liver failure, liver cancer, and premature mortality. Resmetirom is a liver-directed therapy that has demonstrated the potential to treat the liver fibrosis that is associated with these negative outcomes, while resolving the underlying steatohepatitis that drives the disease. The FDA’s acceptance of our NDA with priority review is an important step forward as we pursue our goal of delivering the first approved treatment to patients with NASH with liver fibrosis.”
The FDA grants Priority Review to applications for medicines that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions. A Priority Review designation means FDA’s goal is to take action on an application within 6 months (compared to 10 months under standard review).
Becky Taub, M.D., Chief Medical Officer and President of Research & Development of Madrigal, stated, “We believe that we have delivered a compelling data package to support the FDA’s benefit-risk evaluation of resmetirom for the treatment of NASH with liver fibrosis. The NDA is supported by the positive efficacy results observed in our pivotal Phase 3 trial, the large safety database we have established through the MAESTRO program, and two ongoing outcomes studies that are designed to verify clinical benefit following a potential accelerated approval. We look forward to beginning this critical next phase of the review process.”
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