The US Food and Drug Administration (FDA) has accepted the supplemental New Drug Application (sNDA) for Ocaliva® (obeticholic acid) for the treatment of primary biliary cholangitis (PBC), according to an announcement by Intercept, the manufacturer of the therapy.
The sNDA is intended to fulfill post-marketing requirements, in order to confirm clinical benefits associated with Ocaliva for the treatment of PBC.
Data from the postmarketing COBALT and Study 401 analyses, along with real-world evidence from a claims database and patient registries, were included in the submission package. A Prescription Drug User Fee Act (PDUFA) target action date of October 15, 2024, was assigned by the FDA, with an Advisory Committee meeting being scheduled in the future to discuss the sNDA.
According to Paul Nitschmann, MD, Senior Vice President of Regulatory Affairs at Intercept, “The sNDA data submission asserted that, with more than [7] years on the market and more than 40,000 patient-years of [post-marketing] experience in PBC, Ocaliva is the only second-line therapy that has multiple published analyses of registry data and real-world evidence showing significant effect on transplant-free survival and decompensation-free survival in people living with PBC.”
In 2016, Ocaliva was granted accelerated approval. The agent, which is a farnesoid X receptor (FXR) agonist, is indicated for the treatment of adults with PBC without cirrhosis or for those with compensated cirrhosis who do not have evidence of portal hypertension, either in combination with ursodeoxycholic acid (UDCA) or as monotherapy in those patients who are unable to tolerate UDCA.
Read more about PBC signs and symptoms
The Ocaliva indication is for accelerated approval based on a reduction in alkaline phosphatase (ALP) level. “An improvement in survival or disease-related symptoms has not been established.” In fact, continued approval for this indication may be contingent on verification and a description of the clinical benefit of the agent in confirmatory studies.
PBC is recognized as a progressive, rare, chronic autoimmune disorder that affects the hepatic bile ducts. The disease is most commonly reported in women >40 years of age, with a prevalence of around 1 in 10,000 women. In individuals with PBC, there is a buildup of bile acid in the liver, which leads to inflammation and fibrosis (ie, scarring). If left untreated, these symptoms can result in cirrhosis, liver transplantation, or even death.
“We are pleased that the FDA has filed the submission [for Ocaliva] and look forward to continued, constructive interactions with the agency, including discussing our precedent-setting application during the forthcoming Advisor Committee meeting,” Dr. Nitschmann concluded.
