In another blow to Intercept, the FDA is investigating Ocaliva for potential risk of liver disorder
Just a month before handing Intercept Pharmaceuticals a CRL for Ocaliva in NASH, the FDA began evaluating the drug for a potential risk of liver disorder in primary biliary cholangitis (PBC) patients. The probe, which launched in May, was disclosed by the company deep down in their latest quarterly report filed with the SEC, and had not otherwise been communicated publicly by Intercept until reports surfaced this week.
The FDA has notified us that in the course of its routine safety surveillance, in May 2020 the FDA began to evaluate a newly identified safety signal regarding liver disorder for Ocaliva which the FDA classified as a potential risk.
Ocaliva was approved in 2016 to treat PBC, a chronic disease that affects the liver’s bile ducts. Intercept spokesman Christopher Frates said the biotech is working with the FDA on what will likely be a 12-month evaluation.
An FDA spokesperson declined to offer any additional information aside from what was posted in FAERS, the agency’s adverse event reporting system, which states they are “evaluating the need for regulatory action.”
The probe is the latest blow to Intercept in the last few months. In June, the FDA rejected its NDA for obeticholic acid, the active ingredient in Ocaliva, for the treatment of NASH. Intercept said regulators wanted longer term data from their Phase III trial to back the surrogate endpoint, reduction in liver fibrosis. Company executives argued then that they were blindsided.
CEO Mark Pruzanski had said in a statement:
At no point during the review did the FDA communicate that OCA was not approvable on an accelerated basis, and we strongly believe that the totality of data submitted to date both meet the requirements of the Agency’s own guidance and clearly support the positive benefit-risk profile of OCA.
In a September SEC filing, Intercept announced that 170 jobs were on the chopping block, equal to roughly 25% of its staff, in order to save cash for its continued pursuit of NASH approval. Intercept shares were down 8% yesterday when news of the FDA probe became widely known. An investor on Twitter first noticed the buried disclosure.
Back in 2018, the FDA slapped Ocaliva with a black box warning, due to the drug being “incorrectly dosed” daily instead of weekly. “To ensure correct dosing and reduce the risk of liver problems, we are clarifying the current recommendations for screening, dosing, monitoring, and managing PBC patients with moderate to severe liver disease taking Ocaliva,” it announced.