Intercept files its 'breakthrough' liver drug for rare disease approval

Intercept Pharmaceuticals ($ICPT) has submitted its top candidate to the FDA for approval in primary biliary cirrhosis, a rare disease that results in serious liver damage. The agency generally accepts such applications within 60 days, and, thanks to the FDA's fast-track program, Intercept is eligible for a program that could shorten the standard 10-month review process that ensues. At the same time, the European Medicines Agency has accepted Intercept's application with the same drug, setting in motion a 120-day process. Intercept's drug, obeticholic acid (OCA), is considered a possible blockbuster in large part because of its potential in NASH, a much more common liver ailment with no approved treatments. OCA has shown promise in mid-stage studies on NASH, leading the FDA to confer its coveted breakthrough-therapy designation on the project, and Intercept is in the process of designing a Phase III trial in that indication. More (PDF)

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