Today the FDA announced that it had declined to approve Ocaliva for treatment of NASH. Intercept Pharmaceuticals was set to be the first FDA approved NASH treatment, despite relatively mediocre Phase 3 Results announced in late 2019. This is a major disappointment for the tens of millions of NASH patients that were hoping for an imminent treatment to be available.
The FDA’s response stated the “predicted benefit of OCA…remains uncertain and does not sufficiently outweigh the potential risks to support accelerated approval for the treatment of patients with liver fibrosis due to NASH.” In a sign that there may be future hopes of approval, they suggested that Intercept “submit additional post-interim analysis efficacy and safety data from the ongoing REGENERATE study…and that the long-term outcomes phase of the study should continue.”
So essentially there is no chance of FDA approval for OCA in 2020, but it potentially could still see a reprieve sometime in 2021 or beyond. This has investors running for the exits (stock is currently down 40% today), as the biggest advantage Intercept had was that it would have been the first approved treatment. The actual Phase 3 results were largely unimpressive, especially compared to other drugs in development, but any treatment is better than no treatment. By the time OCA eventually does receive approval, competitors with better treatments may already have beaten them.
In any case, the search for a treatment for NASH goes on. In the meantime, we can all search for ways to make lifestyle changes and eat healthier.