- For the second time, the Food and Drug Administration has delayed an advisory committee meeting to consider approval of Intercept Pharmaceuticals’ drug obeticholic acid (OCA) in the liver disease called NASH. The company says regulators want more data before the meeting.
- The meeting was scheduled for June 9, less than three weeks before the FDA’s approval decision deadline. Postponement of the advisory committee will almost certainly require the agency to delay its decision.
- Intercept wants to expand use of OCA from its current authorization in the rare condition primary biliary cholangitis, in which it is marketed as Ocaliva, to NASH, which affects as many as 15 million U.S. adults. Intercept shares fell 15% to trade at $78.18 in Friday morning trading.
In the race to gain approval for a drug to treat NASH, or non-alcoholic steatohepatitis, Intercept has always been at the head of the pack, ever since the National Institutes of Health-led FLINT trial turned up a surprise positive finding six years ago.
Because so many rivals have suffered setbacks, that leading position has not changed. The latest delay won't necessarily allow others to gain an upper hand either.
Still, the decision to delay the advisory committee meeting has led to "increased uncertainty and lack of clarity for investors," Jefferies analyst Michael Yee wrote in a note to clients.
Intercept chalked up the delay to “review of additional data requested by the FDA that the company intends to submit within the next week." However, the company tried to put a positive light on it, with CEO Mark Pruzanski claiming that the "additional data being submitted will be important in facilitating a more informed discussion" at the advisory panel.
The company acknowledged that the review will probably delay an approval decision, which had been scheduled to occur by June 26.
Yee added that the FDA review guidelines typically don’t allow for two major amendments to a company's data package and two delays to the decision deadline. The approval had already been pushed back from March 26 because of an amendment.
The first delay to the meeting, from April 22 to June 9, had occurred because of the coronavirus pandemic.
Intercept’s application in NASH is based on the results of the REGENERATE trial, in which 18% of patients taking 10 milligram doses of OCA and 23% taking 25 milligram doses saw a one-stage improvement of liver scarring without any worsening of their disease, compared with 12% taking a placebo.
Others have not been so successful. Most recently, the French biopharma company GenFit had a setback in NASH, when its drug elafibranor failed to do better than placebo using similar study goals.