In a Friday disclosure, Intercept Pharmaceuticals said it could take up to three months longer for regulators to make an approval decision on the company's closely watched liver disease drug. The approval deadline is now set for June 26 rather than March 26.
Intercept expected such a delay due to the scheduling of an upcoming Food and Drug Administration meeting. Slotted for April 22, the meeting will have a panel of independent experts evaluate Intercept's drug as a treatment for the disease, and then recommend to the FDA whether its safety and efficacy support approval. Given the timing, it was fairly certain an approval decision would be pushed back until after the panel makes its recommendation.
Despite the delay, Intercept CEO Mark Pruzanski maintains that his company's drug, called obeticholic acid, can enter the U.S. market before the end of June.
"I feel very confident that we will be ready and we will be able to drive the successful launch of this drug," Pruzanski told BioPharma Dive during an interview Wednesday, right before Intercept was notified of the new approval deadline. The company confirmed Friday that it's still guiding for a U.S. approval and launch in the first half of 2020.
Intercept's drug has already gotten the FDA's blessing for a separate condition called primary biliary cholangitis, for which it is marketed as Ocaliva.
If approved again, it would be the first commercial treatment for non-alcoholic steatohepatitis, a fatty liver illness estimated to affect millions of U.S. patients. The large market opportunity has made NASH a crowded area of drug development, with big players such as Pfizer, Novartis and Gilead investing heavily.
Intercept, however, is the only one to have succeeded in a late-stage clinical trial. Results showed that NASH patients with moderate to advanced liver scarring who were put on Intercept's drug ended up experiencing a one-stage-or-greater improvement in fibrosis without their disease worsening.
This effect, though, was seen in just a quarter of patients taking the higher dose of Intercept's drug. Half of all the patients on that dose also reported itching, which analysts worry may limit the drug commercially because patients won't be able to tolerate the side effects. Notably, NASH is considered a "silent" disease because its symptoms are mild or go unnoticed until the later stages, at which point patients have significant enough tissue damage that some require liver transplants.
Pruzanski doesn't agree that NASH is silent. He said these patients often report lower quality of life due to chronic fatigue, abdominal discomfort and the itching that's common with chronic liver disease. While these reports could be affected by other factors — many NASH patients are overweight or diabetic, for example — for Intercept they affirm a desire for more treatment options among patients and doctors.
Gauging patient interest is just one of the challenges Intercept inherits as a first-mover in NASH. The New York-based company also faces an insurance system expected to be very sensitive about price because of the lofty price tags put on medicines for the last big liver disease, hepatitis C.
"There's been a lot of fearmongering out there, with this 'enormous' population, this 'tsunami' of disease," Pruzanski said, "invoking the days of hep C with Sovaldi and other [direct-acting antiviral] launches."
"What we've set out to do is really reassure payers that while this is a large unmet need," he added, "we are appropriately focused on this subset of patients with advanced fibrosis."
Upon launch, Intercept will target the roughly half a million patients who the company expects have advanced fibrosis and are under specialist care. The company has already done more than 100 payer interviews, and internal market research has found 84% of commercial payers see patients with advanced fibrosis patients as the most suitable group to receive its drug.
Yet while Intercept remains bullish on its own prospects, investor confidence in the field cooled last year as Gilead's lead program disappointed.
"Gilead is sort of the bellwether for liver diseases. And so, to the extent they struggle, it doesn't bode well for some of the smaller players," Brian Lian, CEO of NASH drug developer Viking Therapeutics, said in an interview with BioPharma Dive this week.
The year ahead, however, promises several data readouts that could reinvigorate shareholders.
"There was a turnoff in the investment community from these negative studies," Madrigal CEO Paul Friedman told BioPharma Dive in a recent interview. "I think there's been a little bit of a swing back of late, but ... there's a lot of variability."