On March 14, the FDA approved the nation's first medication to treat noncirrhotic non-alcoholic steatohepatitis with fibrosis, or fatty liver scarring.
Also known as MASH, or metabolic dysfunction-associated steatohepatitis, the condition is associated with other health conditions, including high blood pressure and Type 2 diabetes. Federal estimates place the prevalence between 6 million and 8 million people, which is expected to increase.
Madrigal Pharmaceuticals' Rezdiffra (resmetirom) is indicated for MASH adult patients with moderate to advanced liver scarring to be used along with diet and exercise.
In a trial, between 24% and 36% of study participants experienced MASH resolution and did not see worsened liver scarring, compared to between 9% and 13% of those who received a placebo alongside diet and exercise. The range in results is because of different pathologists' readings after 12 months, the FDA said.
"The accelerated approval of Rezdiffra is a culmination of more than 15 years of research from our founder Dr. Becky Taub" — who is also the company's chief medical officer — "and a small R&D team that took on one of the biggest challenges in drug development," Bill Sibold, CEO of Madrigal, said in a news release. "This is a historic moment for the [non-alcoholic steatohepatitis] field and represents the best of what our industry is capable of."
Rezdiffra is greenlit through the agency's accelerator approval, and an ongoing 54-month trial is further evaluating efficacy and safety.