|Intercept CEO Mark Pruzanski|
Intercept Pharmaceuticals ($ICPT) has nabbed the FDA's breakthrough therapy designation (BTD) for obeticholic acid (OCA), a closely watched therapy in the pipeline for a blockbuster NASH indication.
The biotech says the BTD win was based on data from a pair of midstage studies of OCA, which persuaded some analysts to tap it as a major new therapy in the clinic for a rapidly growing population of patients suffering from liver disease. While no guarantee of success, the agency has been handing out BTDs as a hall pass for companies looking for quick access and feedback from the agency. In this case, the company says a breakthrough designation should help grease the tracks for the late-stage program.
The biotech's shares were up a whopping 38% this morning, the latest twist in the stock's wild roller coaster ride.
Intercept is widely viewed as a leader in the field, with a set of stellar results on efficacy dogged by some doubts about troubling side effects. And other top biotechs haven't ignored the potential. Gilead ($GILD) jumped in recently with a $470 million deal with Phenex. Shire ($SHPG) jumped earlier, buying Lumena for up to $260 million. And Enanta was one of the most recent to get started on NASH, with additional programs underway at La Jolla Pharmaceuticals and Raptor.
Nonalcoholic steatohepatitis--NASH--is also called fatty liver disease, a condition characterized by liver inflammation that can lead to fibrosis and possible organ failure. And it's begun spurring havoc in a big group of patients.
"We are very pleased to have received breakthrough therapy designation for NASH with liver fibrosis, as it will enable us to work closely with FDA to finalize the design of our Phase III program," said Dr. Mark Pruzanski, the CEO of Intercept.
- here's the release
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