CONSHOHOCKEN, Pa. — Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a clinical-stage biopharmaceutical company pursuing novel therapeutics for nonalcoholic steatohepatitis (NASH), today announced that the Company’s Marketing Authorization Application (MAA) for resmetirom for the treatment of NASH/metabolic dysfunction-associated steatohepatitis (MASH) with liver fibrosis has been validated and is now under evaluation with the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).
NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally. Resmetirom is a once-daily, oral, liver-directed THR-β agonist designed to target key underlying causes of NASH. The clinical development program for resmetirom is comprised of 18 clinical studies supporting the MAA: twelve Phase 1 studies, two Phase 2 studies, and four Phase 3 studies. The pivotal MAESTRO-NASH trial of resmetirom is the only Phase 3 trial in NASH to achieve both fibrosis reduction and NASH resolution primary endpoints, an efficacy standard for NASH therapies described in the EMA’s Reflection Paper on Regulatory Requirements for the Development of Medicinal Products for Chronic Non-infectious Liver Diseases.
“NASH with fibrosis represents a serious burden for both patients and health systems. Without treatment, the disease can lead to cirrhosis, liver failure, liver cancer and premature death,” said Becky Taub, M.D., Chief Medical Officer and President of Research & Development of Madrigal. “Based on the positive results from our Phase 3 MAESTRO trials, we believe resmetirom has the potential to become the first therapy for patients with NASH with liver fibrosis to receive approval in Europe.”
In the United States, resmetirom received Breakthrough Therapy designation from the FDA and was granted Priority Review with a Prescription Drug User Fee Act (PDUFA) date of March 14, 2024, the target date by which FDA intends to complete its review.
“As we near an FDA decision on accelerated approval of resmetirom in the U.S. and advance our application in Europe, Madrigal is generating new momentum for the NASH field,” said Bill Sibold, Chief Executive Officer of Madrigal. “The results from our pivotal Phase 3 trial recently published in the New England Journal of Medicine provide a robust dataset to support regulatory evaluation globally, and our two ongoing outcomes trials carry the potential to reinforce Madrigal’s long-term leadership position in NASH. Our regulatory, R&D and commercial activities continue to accelerate, and each day brings us one step closer to delivering the first foundational therapy to patients with this serious disease.”
About NASH
Nonalcoholic steatohepatitis (NASH) is a more advanced form of nonalcoholic fatty liver disease (NAFLD). NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally. Additionally, patients with NASH, especially those with more advanced metabolic risk factors (hypertension, concomitant type 2 diabetes), are at increased risk for adverse cardiovascular events and increased morbidity and mortality.
Once patients progress to NASH with significant fibrosis (consistent with fibrosis stages 2 and 3), the risk of adverse liver outcomes increases dramatically. NASH is rapidly becoming the leading cause of liver transplantation in the U.S.
Madrigal estimates that approximately 1.5 million patients have been diagnosed with NASH in the U.S., of which approximately 525,000 have NASH with significant fibrosis. Madrigal plans to focus on approximately 315,000 diagnosed patients with NASH with significant fibrosis under the care of the liver specialist physicians during the launch of resmetirom.
There are currently no FDA-approved therapies available for the treatment of NASH.
NASH is also known as “metabolic dysfunction-associated steatohepatitis (MASH)” following a change in disease nomenclature introduced by hepatology medical societies in 2023.
About Madrigal Pharmaceuticals
Madrigal Pharmaceuticals, Inc. (Nasdaq: MDGL) is a clinical-stage biopharmaceutical company pursuing novel therapeutics for nonalcoholic steatohepatitis (NASH), a liver disease with high unmet medical need. Madrigal’s lead candidate, resmetirom, is a once-daily, oral, liver-directed THR-β agonist designed to target key underlying causes of NASH.
Investor Contact
Tina Ventura
Media Contact
Christopher Frates