FDA approves West Conshohocken firm’s drug for liver disease that soared amid obesity epidemic

A West Conshohocken biotech firm won FDA approval on Thursday for the first drug aimed at treating a common liver disease that has soared amid the obesity epidemic.

The drug, which Madrigal Pharmaceuticals plans to sell as Rezdiffra, was approved to treat scarring and inflammation caused by abnormal accumulations of fat in the liver.

Sometimes called fatty liver disease, the condition affects millions of Americans, and in severe cases it can result in liver cancer or the need for a liver transplant. To start, the drug maker aims to offer the treatment to an estimated 315,000 people with moderate to advanced scarring.

The FDA granted accelerated approval to the drug on the strength of an ongoing, late-stage clinical trial that Madrigal funded, in which the treatment reduced scarring or inflammation in one-quarter to one-third of patients. Other participants’ conditions did not improve significantly during the initial 12 months of the study, illustrating the challenge in addressing a disease with a complex mix of environmental and hereditary causes.

Yet the drug will nevertheless be welcome after repeated unsuccessful attempts by other drug makers, said Yedidya Saiman, a hepatologist at Temple Health and an assistant professor at Temple’s Katz School of Medicine.

“It’s been a long time coming,” said Saiman, who was not involved with the study. “There’s been dozens of candidate drugs that have failed over the years.”

Until now, physicians have treated the condition primarily by counseling patients to lose weight through a combination of diet and exercise, an approach that fails for many, Saiman said.

Some patients also can improve their liver health by treating related metabolic conditions such as type-2 diabetes, the Temple physician said. And researchers now are studying whether liver disease can be treated with drugs in the same class as Ozempic, which are designed to treat diabetes and weight loss.

But Rezdiffra, which has the scientific name of resmetirom, is the first approved drug that specifically targets this liver disease, said Nikolay Nikolov, acting director of the Office of Immunology and Inflammation in the FDA’s Center for Drug Evaluation and Research.

“Today’s approval of Rezdiffra will, for the first time, provide a treatment option for these patients, in addition to diet and exercise,” he said in an FDA news release.

In a company news release, Madrigal chief executive officer Bill Sibold credited the scientists who developed the drug.

“The accelerated approval of Rezdiffra is a culmination of more than 15 years of research from our founder Dr. Becky Taub and a small R&D team that took on one of the biggest challenges in drug development,” he said.

Analysts have estimated that the drug will cost between $40,000 and $50,000 per year, according to STAT, a media outlet that focuses on medicine and life sciences. The treatment would be cost-effective at a price up to $50,000, STAT said, citing an analysis by the nonprofit Institute for Clinical and Economic Review.

The formal name of the condition for which the drug was approved is metabolic dysfunction-associated steatohepatitis (MASH). Until recently, the condition was known as NASH, for non-alcoholic steatohepatitis, to distinguish these patients from those with similar symptoms in which excessive alcohol use played a role.

Liver specialists switched to the new name because it clearly defines what is happening in these patients, rather than defining the disease by something that is not present (excessive alcohol use). The name is so new that even the FDA used the old one in its announcement on Thursday.

At least 1.5 million Americans have been diagnosed with MASH, company officials said. Initially, the drug will be aimed at an estimated 315,000 patients who have moderate to advanced scarring in the liver.

Rezdiffra works by activating a receptor on a type of hormone found in the liver, which in turn reduces the amount of fat in that organ. Side effects can include diarrhea and nausea.

Diet and exercise will continue to be a key element of managing the liver disease, Temple’s Saiman said.

Madrigal was founded in 2011 and went public in 2016 in the process of merging with Massachusetts-based Synta Pharmaceuticals Corp.