FDA Greenlights Madrigal's Rezdiffra, Pioneering NASH Treatment
The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Madrigal Pharmaceuticals' Rezdiffra (resmetirom) for the treatment of adults with non-cirrhotic nonalcoholic steatohepatitis (NASH) with moderate to advanced liver fibrosis. This marks the first FDA approval for a medication to treat this condition, which is a severe form of fatty liver disease. The decision was informed by data from the Phase 3 MAESTRO-NASH trial, which demonstrated that Rezdiffra could improve liver fibrosis and resolve NASH in the targeted patient population.
Rezdiffra works as a partial activator of the thyroid hormone receptor, aimed at reducing liver fat accumulation. The drug is recommended for use alongside diet and exercise, with the prescribed dosage varying based on body weight. However, the FDA has also released warnings regarding the potential for drug-induced liver toxicity and gallbladder-related issues, cautioning against its use with cholesterol-lowering statins and certain other medications.
The approval under the FDA's accelerated pathway indicates that Rezdiffra addresses a significant unmet medical need. It comes with a requirement for Madrigal Pharmaceuticals to conduct further post-approval studies to confirm the drug's clinical benefit. In the meantime, the approval enables the company to begin marketing Rezdiffra in the U.S., with an expected significant market potential. Analysts predict substantial sales, with GlobalData forecasting revenues of $1 billion globally by 2027. The drug's annual wholesale acquisition cost (WAC) is set at $47,400. Further regulatory decisions, such as the European Medicines Agency's (EMA) review, are pending for Rezdiffra's approval in other markets.
