WASHINGTON – The United States Food and Drug Administration (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 multibillion-dollar opportunity.
The company’s shares surged 24 per cent to US$301.99 in extended trading.
Nash, which was recently renamed metabolic dysfunction-associated steatohepatitis (Mash), affects about 1.5 million people in the US, according to the company’s estimates, but there was no approved treatment for it until now.
Madrigal’s oral drug will be available under the brand name Rezdiffra from April, chief executive Bill Sibold told Reuters before the approval.
The company has priced the drug at an annual wholesale price of US$47,400 (S$63,000).
The Institute for Clinical and Economic Review, a drug pricing watchdog, in May 2023 estimated Madrigal’s drug would meet the cost-to-effectiveness benchmark if it was priced between US$39,600 and US$50,100 per year.
Evercore International Strategy and Investment analyst Liisa Bayko estimates Rezdiffra’s annual sales to surpass US$5 billion at its peak.
Madrigal’s drug has been approved for patients who have Nash with fibrosis, or scarring, that has progressed to stage two or three in severity, the company said on March 14.
But the FDA’s label, does not require a liver biopsy requirement for diagnosis of Nash, the company added, which was a key concern among experts that could have hit demand.
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 of the organ.
The approval marks a “game-changing” moment for patients after a string of failures over the last decade, said Ms Lorraine Stiehl, CEO of patient advocacy organisation American Liver Foundation.
She said: “There are a lot of skeletons, there are a lot of tombstones in terms of therapies in the space.
“(But) the... approval is going to open the floodgates to just so many more therapeutics, which are desperately needed.”
Other biotech firms, such as Akero Therapeutics, 89bio, Viking Therapeutics and Sagimet Biosciences, are conducting middle and late-stage trials of their drugs.
Rezdiffra’s accelerated approval was based on data from a late-stage study of 888 patients, which showed those on the drug had significant resolution of symptoms or improvement in liver scarring as compared with those on placebo, according to the FDA. REUTERS
