The fatty liver disease market has seen a thinning pipeline of late, but analysts at GlobalData still forecast a lucrative market for what is a growing healthcare problem.
Nonalcoholic steatohepatitis (NASH) is an advanced form of a fatty liver characterized by inflammation of the organ and is caused, predominately by obesity, with Type 2 diabetes also a known risk factor.
It’s tough to diagnose, however, and is known as a “silent disease” as there are few to no symptoms of NASH until it gets to a later stage, which can cause more serious damage and scarring to the liver.
Its prevalence is growing, with the Liver Foundation estimating that about 5% of all Americans have the disease.
Pharma has long seen this as a major drug market opportunity, but there remain no NASH drugs despite years of research and trials. This is because clearing fat from the liver efficiently and safely has proven to be much more difficult than first thought, with the NASH pipeline littered with flops and setbacks.
The most recent failure came in the form of a complete response letter from the FDA for Intercept Pharmaceuticals' farnesoid X receptor agonist Ocaliva. This has an FDA approval in a certain type of liver disease, but the FDA has now twice rejected its NASH license, more recently because of the drug’s poor efficacy and questions over its safety.
GlobalData suggests that despite the setbacks and Intercept's failure, previously the most advanced NASH player but now essentially withdrawing, the fatty liver market still "holds enormous untapped potential." The focus now shifts to other pipeline hopefuls, seen as the new potential saviors of this emerging market.
These include Madrigal Pharmaceuticals' resmetirom, a selective thyroid hormone receptor-β agonist, which GlobalData analysts predict “will most likely be the first drug candidate approved for NASH in both the U.S. and EU.”
The future, however, may also come in the form of combos. “To increase their chance of success in the NASH market, established companies may look to develop combination therapies to target multiple stages of NASH progression to develop a successful treatment,” the GlobalData analysts said in a release.
They note that diabetes specialist Novo Nordisk is already working on using its franchise semaglutide (the main ingredient in Wegovy, Rybelsus and Ozempic) in tandem with Gilead Sciences' cilofexor and firsocostat in more advanced forms of NASH.