In this illustration, the Madrigal Pharmaceuticals logo is displayed on a smartphone screen.
Rafael Henrique | SOPA Photos | Rocket Lite | Getty Images
The U.S. Food and Drug Administration on Thursday approved the first-ever treatment for a common and fatal form of liver disease that affects millions around the world.
Meaning the decision of the Food and Drug Administration Madrigal Pharmaceuticals It has succeeded in a disease space that many large companies have failed – or are still trying to break into. Madrigal shares jumped more than 20% in extended trading Thursday after the approval.
Novo Nordisk And Eli Lilly They are testing their popular weight-loss injections as treatments for the same type of liver disease, called nonalcoholic steatohepatitis, or NASH.
Madrigal, which will be marketed as Rezdiffra, is specifically approved to treat patients with non-alcoholic steatohepatitis who also have moderate to advanced liver scarring. The treatment must be used in combination with diet and exercise, according to the US Food and Drug Administration.
Non-alcoholic steatohepatitis (NASH) is a serious form of liver disease characterized by excess fat accumulation and inflammation in the liver and can lead to liver scarring, also known as fibrosis, along with liver failure and liver cancer. This condition is often associated with other health problems, such as high blood pressure, type 2 diabetes and obesity.
Approximately 6 million to 8 million people in the United States have nonalcoholic steatohepatitis with moderate to advanced liver scarring, according to an estimate reported by the U.S. Food and Drug Administration.
Madrigal said in a statement that the drug will be available in April. The company also said it has created an assistance program to help people without insurance access Rezdiffra. Madrigal did not disclose the cost of treatment.
“Previously, patients with non-alcoholic steatohepatitis who also had significant scarring of the liver did not have a drug that could directly address liver damage,” said Dr. Nikolai Nikolov, acting director of the FDA's Office of Immunology and Inflammation.
Madrigal specifically has received “accelerated approval” from the Food and Drug Administration. This classification clears drugs more quickly if they meet an unmet medical need for serious conditions, and requires the drug maker to further study the treatment and verify its clinical benefits.
Madrigal works by activating the thyroid hormone receptor in the liver to help reduce fat accumulation. Patients take it orally every day.
In a recent study published last month, Rezdiffra helped resolve symptoms of nonalcoholic steatohepatitis and improve liver scarring without worsening the condition. It is worth noting that the rate of serious adverse events was similar between the group of patients who took the drug and the group who received a placebo.
The most common side effects associated with treatment were diarrhea, nausea, and vomiting.
Some specialists have begun calling metabolic dysregulation-associated steatohepatitis NASH, or MASH, to avoid potentially stigmatizing language.
