Why Aren't There Any FDA-Approved Drugs To Treat NASH?

By Jennifer Dennis-Wall, Ph.D.

The liver does many important things in the body, such as filtering of the blood and metabolizing all compounds, including food and drugs, that enter the body. In that vein, liver disease can be absolutely devastating.

Non-alcoholic fatty liver disease (NAFLD) affects almost 100 million, or around 25%, of people in the US alone, and the economic burden is over $100 billion annually, yet treatments are surprisingly limited. Treatment focuses mostly on reducing body weight, since obesity and metabolic syndrome are strongly associated with NAFLD, or resorting to a liver transplant. Those are not very good options, since weight loss is tough to achieve and maintain while liver transplants can mean serious wait time and lifelong immunosuppression afterwards. Without reversing the NAFLD fat accumulation in the liver, progression to non-alcoholic steatohepatitis (NASH) is a real and fatal possibility.

No drugs have been approved by the FDA to treat NAFLD or NASH. The only pharmaceuticals currently used to treat the diseases are geared more towards improving the comorbid insulin resistance that most patients have. For such a serious, chronic disease affecting so many people, that is simply unacceptable. Fortunately, there are some promising studies being conducted right now with numerous companies invested in the race. According to BioPharma Dive’s estimates, there are about 50 NASH candidates in phase I-III trials. Of course, only a small percentage of these, if any, will move forward in the pipeline.

Why is there such a large gap to fill in this indication?