According to a press release from the US Food and Drug Administration (FDA), the agency has approved the medication resmetirom (marketed as Rezdiffra) as the first-ever treatment intended to treat liver scarring in adults living with noncirrhotic nonalcoholic steatohepatitis (NASH), also called metabolic dysfunction-associated steatohepatitis (MASH). This treatment is intended to address moderate to advanced scarring (fibrosis) of the liver, in tandem with lifestyle changes to exercise and diet.
In the past, patients living with this serious disease lacked a medication that could directly treat fibrosis of the liver. Rezdiffra is classified as a partial activator of thyroid hormone receptor. This mechanism reduces the buildup of fat in the liver, slowing progression of liver damage in the disease.
About the Drug
This approval follows the evaluation of a surrogate endpoint at the 12 month point in a 54-month trial. This endpoint measured the extent of liver scarring and inflammation and suggests clinical benefit. This approval using a surrogate endpoint means that Rezdiffra was approved under Accelerated Approval protocol. This means that the developing company will need to continue evaluating the drug post-approval in order to fully demonstrate clinical benefit. If it fails to do so, then the approval of Rezdiffra would be revoked.
About the Trial
888 patients participated in the study, with 294 people receiving placebo, 298 receiving 80 mg of the drug, and 296 receiving 100 mg of the drug, alongside standard-of-care treatment for NASH. Following 12 months of this treatment, biopsy of the liver indicated that up to 27% of patients who received the 80 mg dose and up to 36% of patients who got the 100 mg dose saw no further progression of liver scarring or resolution of the disease. Meanwhile, up to 13% of the placebo group experienced these outcomes. Meanwhile 23% of patients on the 80 mg dose saw no worsening of disease and improvement in fibrosis, and 28% using the 100 mg dose experienced the same effects, compared to just 15% of the placebo group.
About Nonalcoholic Steatohepatitis (NASH)
Nonalcoholic steatohepatitis (NASH), also called metabolic dysfunction-associated steatohepatitis, is a type of liver disease in which fat is deposited in the liver independent of excessive alcohol consumption. This disease can progress rapidly. Risk factors include metabolic syndrome and insulin resistance. There also appears to be some genetic component to the disease as well. This condition also increases the risk of other health problems and liver cancer. Men also seem to be at greater risk, getting the disease as almost twice the rate that women do. Symptoms of nonalcoholic steatohepatitis include jaundice, malaise, fatigue, and abdominal pain or discomfort. Without treatment, the liver can become scarred, and the patient may need a liver transplant. However, the condition can also be managed with proper diet, the use of certain medications, and exercise. To learn more about nonalcoholic steatohepatitis, click here.
