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-ever 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 pharmaceutical products has succeeded in an area of the disease that several larger companies have failed – or are still trying to break into. Madrigal shares rose more than 20% in extended trading Thursday following the approval.
Novo Nordisk AND Eli Lilly are testing their respective blockbuster 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 must be used with diet and exercise, according to the FDA.
NASH is a severe form of liver disease characterized by accumulation of excess fat 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 hypertension, 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 will be available in April. The company also said it has established an assistance program to help people who do not have access to Rezdiffra insurance. Madrigal did not reveal how much the treatment will cost.
“Previously, NASH patients who also had significant liver scarring did not have access to a drug that could directly address the 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 clears drugs more quickly if they fill an unmet medical need for serious conditions and requires the pharmaceutical manufacturer to further study the treatment and verify its clinical benefits.
Madrigal’s drug works by activating a thyroid hormone receptor in the liver to help reduce fat buildup. Patients take it orally every day.
In a late-stage study published last month, Rezdiffra helped resolve NASH symptoms and improve liver scarring without worsening the condition. In particular, the rate of serious adverse events was comparable between the group of patients who took the drug and the other group who received a placebo.
The most common treatment-related side effects were diarrhea, nausea and vomiting.
Some specialists have begun calling steatohepatitis associated with metabolic dysfunction NASH, or MASH, to avoid potentially stigmatizing language.