The U.S. FDA has approved Madrigal Pharmaceuticals' drug for a fatty liver disease known as non-alcoholic steatohepatitis (NASH), the first treatment to get the nod for the condition and opening up a multi-billion opportunity.
The company's oral drug, called Rezdiffra, has been approved for patients who have NASH with fibrosis, or scarring, that has progressed to stage 2 or 3 in severity, according to an FDA label released on Thursday.
NASH, which was recently renamed metabolic dysfunction-associated steatohepatitis (MASH), affects about 1.5 million people in the United States, according to the company's estimates, but there was no approved treatment for it until now.
The cause of NASH is not fully understood. It is typically associated with obesity, hypothyroidism, diabetes, high levels of fat in the blood, and similar health issues. It causes excess build up of fat in the liver and causes inflammation and fibrosis, or scarring, of the organ.
Madrigal's drug will be available from April, CEO Bill Sibold told Reuters in an interview before the approval.
The approval marks a "gamechanging" moment for patients after a string of failures over the last decade, said Lorraine Stiehl, CEO of patient advocacy organization American Liver Foundation.
"There's a lot of skeletons, there's a lot of tombstones in terms of therapies in the space," Stiehl said. "(But) the ... approval is going to open the floodgates to just so many more therapeutics which are desperately needed."
Akero Therapeutics, 89bio, Viking Therapeutics and Sagimet Biosciences are conducting mid- and late-stage trials of their drugs.
