The US Food and Drug Administration (FDA) has approved Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) to treat adults with noncirrhotic non-alcoholic steatohepatitis (NASH) with moderate-to-advanced liver fibrosis, alongside diet and exercise.
Based on results from the MAESTRO-NASH trial, the accelerated approval marks Rezdiffra as the first treatment option available to address notable liver scarring and liver damage.
Often associated with other health issues, including high blood pressure and type 2 diabetes, NASH is caused by the progression of non-alcoholic fatty liver disease, where liver inflammation leads to liver scarring and liver dysfunction over time.
NASH, with moderate-to-advanced liver scarring, affects over six million people in the US.
Madrigal’s Rezdiffra is a partial activator of a thyroid hormone receptor, which is used alongside diet and exercise and works to reduce liver fat accumulation.
Involving 1,759 patients with biopsy-confirmed NASH and published in the New England Journal of Medicine, the ongoing pivotal trial showed that over 26% of patients receiving 80mg of Rezdiffra and up to 36% of patients who received 100mg of Rezdiffra experienced NASH resolution with no worsening of liver fibrosis at 12 months, compared to 9% to 13% who received placebo and counselling on diet and exercise.
Furthermore, 23% of patients who received 80mg of Rezdiffra and up to 28% who received 100mg experienced an improvement in liver scarring with no worsening of NASH compared to over 13% of those who received placebo.
“This is a historic moment for the NASH field and represents the best of what our industry is capable of,” said Madrigal’s chief executive officer, Bill Sibold.
“For the first time, [Rezdiffra will] provide a treatment option for these patients, in addition to diet and exercise,” said Nikolay Nikolov, acting director, FDA’s Center for Drug Evaluation and Research, office of immunology and inflammation.
Becky Taub, founder, chief medical officer and president, research and development, Madrigal, commented: “Rezdiffra will change the treatment paradigm for NASH with moderate to advanced liver fibrosis, giving physicians a liver-directed therapy to help improve fibrosis and resolve NASH before their patients progress to cirrhosis.”