The company announced its decision to stop all NASH-related investment and restructure its business in response to the FDA’s decision on Thursday.
The drug developed by Intercept Pharmaceuticals (ICPT.O) to treat a specific form of fatty liver disease was denied accelerated approval by the US Food and Drug Administration on Thursday, sending its shares down more than 11% in extended trading.
The rejection represents Intercept’s second unsuccessful attempt to obtain approval for the drug to treat patients with non-alcoholic steatohepatitis (NASH), a liver disease that affects 5% of U.S. adults but for which there are currently no approved treatments.
The FDA previously rejected Intercept in 2020 because it found that the drug’s anticipated efficacy did not outweigh potential risks.
The company announced its decision to stop all NASH-related investment and restructure its business in response to the FDA’s decision on Thursday. This will allow it to strengthen its focus on rare and serious liver diseases.
According to Intercept, the FDA determined that the drug’s marketing application cannot be approved in its current form.
The FDA’s decision followed a predictable course. In May, its advisory panel suggested delaying the oral drug’s accelerated approval until the results of its late-stage study are available.
Before the May meeting, the health regulator’s staff reviewers had raised a number of safety issues, including drug-induced liver damage and an elevated risk of diabetes linked to the medication, which already has its efficacy called into question.
Since then, analysts have predicted that the company will discontinue its advanced NASH trial and concentrate on increasing its market share in the management of primary biliary cholangitis (PBC), a protracted liver disease.
The first NASH treatment is being developed by a number of pharmaceutical companies, including Novo Nordisk (NOVOb.CO), Madrigal Pharmaceuticals (MDGL.O), and Akero Therapeutics (AKRO.O).
Nonalcoholic steatohepatitis (NASH), a component of nonalcoholic fatty liver disease, is liver inflammation and damage brought on by an accumulation of fat in the liver. Most people don’t experience any symptoms, but some fats can damage liver cells and cause inflammation, which can lead to problems with the liver.