Tokyo, 17 March, /AJMEDIA/
Madrigal Pharmaceuticals Inc.’s drug Rezdiffra gained the first US approval to treat a potentially deadly liver disease that affects millions worldwide, succeeding in an area where some bigger rivals have failed, according to Bloomberg.
The medication, also called resmetirom, gained accelerated Food and Drug Administration approval to treat patients with nonalcoholic steatohepatitis, or NASH, and moderate to advanced liver fibrosis, according to a statement Thursday from West Conshohocken, Pennsylvania-based Madrigal. Patients won’t need an invasive liver biopsy to get the drug, which will cost $47,400 a year, company officials said on a call.
Biopsies involve inserting a needle into the liver to extract a piece of tissue, a procedure patients don’t like. Currently, specialists measure the liver disease with scans or blood tests. The lack of a liver biopsy requirement “removes a potential access barrier for patients and could accelerate patient identification and Rezdiffra adoption,” Andy T. Hsieh, a William Blair analyst, said in a note.
Madrigal shares rose as much as 23% when US markets opened Friday, its biggest increase since December 2022, adding about $1.1 billion in market value. Viking Therapeutics Inc., which is also developing a NASH therapy, rose as much as 4%.
NASH has been diagnosed in some 1.5 million Americans, sometimes leading to permanent liver damage and death. Bristol Myers Squibb Co., Gilead Sciences Inc. and others have tried and failed to develop treatments, and analysts surveyed by Bloomberg estimate annual sales of Madrigal’s might 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 Chief Executive Officer Bill Sibold said in the statement.
NASH is a form of fatty liver disease that can lead to 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 roughly 5% of people worldwide may be affected. Madrigal plans to focus initially on reaching some 315,000 US patients who are being seen by specialists for the disease.
It frequently goes undiagnosed, said Donna Cryer, founder of the Global Liver Institute advocacy group, with patients discovering they have it during gall bladder removal, screening for liver cancer or evaluation for a transplant. The potential for treatment may motivate more doctors to screen earlier for the condition itself, she said.
“This would really open the doors for the liver health field to grow in the same ways as heart health or brain health,” she said in an interview.
Numerous companies have been frustrated in the race to develop drugs to treat the ailment, seen as an attractive multibillion-dollar opportunity. Gilead’s clinical trial failed in 2019. In September, Bristol scrubbed its development program, while Intercept Pharmaceuticals Inc. offloaded its drug after a second rejection from US regulators.
Competition still looms from drugs under development at Akero Therapeutics Inc., Ionis Pharmaceuticals Inc. and other companies. Several obesity drugs are also being studied for their ability to treat NASH, and likely represent the biggest threat to those like Madrigal’s that specifically target the liver disease, according to analysts at Stifel.
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 Review, said its drug would be cost-effective at $39,600 to $50,100 a year.
