In this photo illustration the Madrigal Pharmaceuticals logo seen displayed on a smartphone screen.
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The Food and Drug Administration on Thursday approved the first treatment for a common and potentially deadly form of liver disease that affects millions of people around the world.
The FDA's decision means Madrigal Pharmacy has succeeded in a disease area that several larger companies have failed or are still trying to enter. Madrigal shares rose more than 20% in extended trading Thursday following the approval.
Nordisk and Eli Lilly are testing their respective successful weight-loss injections as treatments for the same type of liver disease, called nonalcoholic steatohepatitis, or NASH.
Madrigal's drug, which will be marketed as Rezdiffra, is specifically approved to treat NASH patients who also have moderate to advanced liver scarring. The treatment should be used with diet and exercise, according to the FDA.
NASH is a severe form of liver disease characterized by excessive fat accumulation and inflammation in the liver and can lead to liver scarring, also known as fibrosis, along with liver failure and liver cancer. The condition is often associated with other health problems, such as high blood pressure, type 2 diabetes and obesity.
About 6 million to 8 million people in the U.S. have NASH with moderate to advanced liver scarring, according to one estimate cited by the FDA.
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 who are uninsured access Rezdiffra. Madrigal has not revealed how much the treatment will cost.
“Previously, patients with NASH who also have notable liver scarring did not have a medication that could directly address liver damage,” said Dr. Nikolay Nikolov, acting director of the FDA's Office of Immunology and Inflammation.
Madrigal's drug specifically received “accelerated approval” from the FDA. That designation authorizes drugs more quickly if they meet an unmet medical need for serious conditions and requires Madrigal to further study the treatment and verify its clinical benefits.
MadrigaL's medication works by activating a thyroid hormone receptor in the liver to help reduce fat accumulation. Patients take it orally every day.
In a late-stage study published last month, Rezdiffra helped resolve NASH symptoms and improve liver healing without worsening the condition. In particular, the rate of serious adverse events was comparable between the group of patients who took the drug and another group who received a placebo.
The most common treatment-related side effects were diarrhea, nausea, and vomiting.
Some specialists have begun calling NASH metabolic dysfunction-associated steatohepatitis, or MASH, to avoid potentially stigmatizing language.
