The medicine Rezdiffra from Madrigal Pharmaceuticals won the first approval in the United States to treat a life-threatening liver disease that affects millions of people around the world, succeeding in an area where some larger rivals have failed.
The medicine, also called resmetiromwon accelerated approval from the Food and Drug Administration to treat patients with non-alcoholic steatohepatitisor NASH, and liver fibrosis moderate to advanced, according to a Thursday statement from Madrigal, based in West Conshohocken, Pennsylvania. Patients will not need an invasive liver biopsy to get the drug, which will cost $47,400 a year, company officials said in a call.
Biopsies involve inserting a needle into the liver to remove a piece of tissue, a procedure that patients do not like. Currently, specialists measure liver disease with scans or blood tests. The lack of a liver biopsy requirement “removes a potential barrier to entry for patients and could accelerate patient identification and adoption of Rezdiffra,” William Blair analyst Andy T. Hsieh said in a note.
Madrigal shares rose as much as 23 percent when U.S. markets opened on Friday, their biggest gain since December 2022, adding about $1.1 billion in market value. Viking Therapeutics, which is also developing a therapy for NASH, rose as much as 4 percent.
NASH has been diagnosed in approximately 1.5 million Americans, sometimes leading to permanent liver damage and death. Bristol Myers Squibb, Gilead Sciences and others have tried to develop treatments without success, and analysts surveyed by Bloomberg estimate that Madrigal’s annual sales could exceed $5 billion by 2031.
“This is a historic moment for the NASH field and represents the best of what our industry is capable of,” Madrigal CEO Bill Sibold said in the statement.
NASH is a form of fatty liver disease that can cause fibrosis or scarring and can be life-threatening in severe cases. Some specialists have begun calling it metabolic dysfunction-associated steatohepatitis, or MASH, to avoid potentially stigmatizing language.
While estimates vary widely, a 2023 study found that approximately 5 percent of people worldwide may be affected. Madrigal plans to initially focus on reaching some 315,000 American patients who are being treated by specialists for the disease.
It often goes undiagnosed, said Donna Cryer, founder of the advocacy group Global Liver Institute, and patients discover they have it during gallbladder removal, liver cancer screening or evaluation for a transplant. The treatment potential may motivate more doctors to perform earlier testing to detect the condition itself, she said.
“This would really open the doors for the field of liver health to grow in the same way as heart health or brain health,” he said in an interview.
They investigate more medications to treat obesity
Many companies have been frustrated in the race to develop drugs to treat the disease, seen as an attractive multibillion-dollar opportunity. Gilead’s clinical trial failed in 2019. In September, Bristol canceled its development programwhile Intercept Pharmaceuticals ditched its drug after a second rejection by US regulators.
Competition still looms from drugs in development at Akero Therapeutics, Ionis Pharmaceuticals and other companies. Several obesity drugs are also being studied for their ability to treat NASH and likely pose the greatest threat to those like Madrigal’s that specifically target liver disease, according to Stifel analysts.
Founded in 2011, Madrigal reported results from a late-stage trial showing its drug reduces fat deposits and scarring caused by the disease. Last year, a nonprofit research group, the Institute for Clinical and Economic Reviewsaid his drug would be profitable between $39,600 and $50,100 a year.
