Breaking a Therapeutic Curse

In June, a surprising decision from the FDA dealt the fatty liver disease research community its toughest blow yet. The agency refused to approve Intercept’s Ocaliva, a treatment for nonalcoholic steatohepatitis (NASH) – a condition characterized by liver inflammation and damage. The widely anticipated therapy would have been the first approved to treat the disease, but was rejected due to an uncertain risk-benefit profile. The news came on the heels of disappointing phase III results in May from French biotech Genfit, whose drug, elafibranor, failed to distinguish itself from placebo. NASH is a growing health concern – and these recent failures have sucker-punched the industry. The number of people living with NASH in the US, for example, is predicted to grow from 16.5 million to 27 million over the next 10 years (1) – contributing to a worsening healthcare bill whose direct costs are estimated at US$600 million to $1 billion per year (2). Predictions suggest that NASH will soon replace hepatitis C as the main cause of liver transplants in the US (3). Thus, there is a growing sense of urgency about the disease and its comorbidities.