Intercept ‘disappointed’ after FDA setback for top NASH contender; stock drops 16%
Click Here to Manage Email Alerts
Intercept Pharmaceuticals saw a 16% drop in its stock value after an FDA advisory committee declined to approve obeticholic acid in pre-cirrhotic patients with liver fibrosis due to nonalcoholic steatohepatitis.
The vote before the Gastrointestinal Drug Advisory Committee on May 19 was Intercept’s second bid to put its 25 mg obeticholic acid product (Ocaliva) in a lead position as the first FDA-approved NASH treatment. In 2020, the FDA rejected Intercept’s application for NASH on the grounds that the benefits of obeticholic acid (OCA) did not outweigh potential risks — a sentiment echoed by the FDA panel in their most recent vote.
“We are disappointed in the outcome of [the FDA] meeting,” Jerry Durso, president and CEO of Intercept, said in a press release. “We continue to disagree with the FDA on certain characterizations of OCA’s efficacy and safety in pre-cirrhotic fibrosis due to NASH and the role of non-invasive tests (NITs), as discussed in [the] meeting. The robust body of evidence provided by Intercept was underscored by public testimony from the liver community, who supported OCA as an option to address the urgent treatment need in NASH and the use of NITs to manage this devastating disease in clinical practice.”
The FDA panel voted 12 to 2 with 2 abstentions that the benefits of 25 mg obeticholic acid failed to outweigh its risks among patients with NASH and stage 2 or 3 fibrosis, most notably drug-induced liver injury and quality of life. As with its earlier rejection, OCA’s shortcoming was its “unfavorable benefit-risk profile” according to Ruby Mehta, MD, from the FDA Office of New Drugs.
“In this resubmission, our assessment of efficacy has remained unchanged,” she said. “... Our concerns regarding the safety/risks also remain unchanged. Given these findings, FDA continues to believe that the benefit-risk profile of OCA 25 mg remains concerning.”
During the FDA meeting, Thomas Capozza, MD, FACP, executive director of clinical research at Intercept Pharmaceuticals, disputed the FDA panel’s interpretation of the data.
“Our NASH clinical development program has shown treatment with OCA 25 mg results in clinically meaningful, anti-fibrotic effects,” he said. “The goal of therapy is to slow, halt or reverse disease progression. OCA 25 mg not only meets the primary fibrosis endpoint of reversal at 18 months, but also attenuates fibrosis progression, as well as improves noninvasive tests in patients with no change in fibrosis stage. This anti-fibrotic effect is highly likely to lead to clinical benefit.”
Intercept noted that, although not binding, the FDA often sides with the advisory committees when making their final recommendations. The FDA is scheduled to issue an approval decision by June 22.
References:
- Reuters. Intercept tumbles on concerns over prospects of fatty liver disease drug. https://www.reuters.com/business/healthcare-pharmaceuticals/intercept-shares-tumble-concerns-over-nash-drug-prospects-2023-05-22/. Accessed May 22, 2023.
- ‘Unfavorable benefit-risk’: FDA panel votes against obeticholic acid approval for NASH. https://www.healio.com/news/gastroenterology/20230519/unfavorable-benefitrisk-fda-panel-votes-against-obeticholic-acid-approval-for-nash Published May 19, 2023.
Click Here to Manage Email Alerts