An experimental drug for fatty liver disease met all of its goals in a late-stage study, researchers reported on Friday at the annual meeting of the European Association for the Study of the Liver in Vienna.
In a randomized trial, resmetirom from Madrigal Pharmaceuticals reduced the amount and severity of liver damage compared to a placebo in 966 patients with NASH, or non-alcoholic steatohepatitis. Most patients also had diabetes.
Roughly 25% to 30% of people worldwide have fatty liver disease, and about 25% of those have NASH, study leader Dr. Stephen Harrison of Pinnacle Clinical Research and Summit Clinical Research in San Antonio, Texas, said at a press conference.
In NASH, liver inflammation from fat leads to scarring, or fibrosis, and eventually to cirrhosis, liver failure, liver transplantation or death. Risk factors include obesity, gastric bypass surgery, high cholesterol and type 2 diabetes.
Resmetirom would be the first approved treatment and its impact on clinical practice would be huge, Harrison said.
NASH is likely under-diagnosed because without a way to treat it, doctors are reluctant to test for it in patients without symptoms, he said.
"Now ... we are at the forefront of treatment," and NASH is more likely to be diagnosed and treated before liver failure develops, Harrison said.
After one year of treatment, NASH had been resolved in 27% of those receiving resmetirom and in 10% of those taking placebo, liver biopsies showed.
Scarring severity decreased in roughly 25% of resmetirom-treated patients versus 14% of the placebo group.
The U.S. Food and Drug Administration has designated resmetirom a "breakthrough therapy" and granted it fast-track status in the approval process.
Read about other experimental drugs for fatty liver disease on Reuters.com