In this photo illustration, the Madrigal Pharmaceuticals logo is displayed on a smartphone screen.
Rafael Henrique | SOPA images | Light rocket | Getty Images
The Food and Drug Administration on Thursday approved the first treatment for a common and potentially fatal form of liver disease that affects millions of people worldwide.
The FDA’s decision means Madrigal Pharmaceuticals has succeeded in a disease area where several larger companies have failed – or are still trying to break into. Shares of Madrigal rose more than 20% in extended trading Thursday following the approval.
Novo Nordisk And Eli Lilly are testing their respective blockbuster weight-loss injections to treat the same type of liver disease, called nonalcoholic steatohepatitis, or NASH.
Madrigal’s drug, marketed as Rezdiffra, is specifically approved for the treatment of patients with NASH who also have moderate to advanced liver scarring. According to the FDA, treatment must be combined with diet and exercise.
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 called fibrosis, as well as liver failure and liver cancer. The condition is often accompanied by other health problems such as high blood pressure, type 2 diabetes and obesity.
According to an estimate cited by the FDA, approximately 6 to 8 million people in the United States suffer from NASH with moderate to advanced liver scarring.
Madrigal said in a statement that the drug would be available in April. The company also said it has set up an assistance program to help people without insurance access Rezdiffra. Madrigal did not disclose how much the treatment will cost.
“Until now, patients with NASH who also had significant liver scarring had no medication that could directly address their 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. This designation allows drugs to be released more quickly if they address an unmet medical need for serious diseases and requires the drug maker to further study the treatment and verify its clinical benefit.
Madrigal’s drug activates a thyroid hormone receptor in the liver, helping to reduce fat accumulation. Patients take it orally every day.
In a late-stage study published last month, Rezdiffra helped relieve the symptoms of NASH and improve scarring in the liver without making the condition worse. Notably, 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 to refer to NASH as metabolic dysfunction-associated steatohepatitis, or MASH, to avoid potentially stigmatizing language.
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2024-03-14 23:08:37
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