MORRISTOWN, N.J. — Intercept Pharmaceuticals, Inc., a biopharmaceutical company and wholly owned subsidiary of Alfasigma S.p.A. focused on the development and commercialization of novel therapeutics to treat rare and serious liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for Ocaliva for the treatment of individuals with primary biliary cholangitis (PBC). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of October 15, 2024. In this communication, the agency informed Intercept that they are planning to hold an Advisory Committee meeting to discuss the application.
“The sNDA data submission asserted that, with more than seven 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 and decompensation-free survival in people living with PBC,” said Paul Nitschmann, M.D., Senior Vice President of Regulatory Affairs at Intercept. “We are pleased that the FDA has filed the submission and look forward to continued, constructive interactions with the agency, including discussing our precedent-setting application during the forthcoming Advisory Committee meeting.”
Ocaliva received accelerated approval in 2016 and is indicated for the treatment of adult patients living with primary biliary cholangitis (PBC) without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension, either in combination with ursodeoxycholic acid (UDCA) with an inadequate response to UDCA or as monotherapy in patients unable to tolerate UDCA. The precedent-setting sNDA for Ocaliva is intended to satisfy the post-marketing requirements to confirm a clinical benefit in patients with PBC. The sNDA is supported by data from the Company’s post-marketing requirement studies COBALT and Study 401 as well as real-world evidence from a U.S. claims database and international PBC patient registries. Intercept and Alfasigma remain committed to supporting people living with PBC and look forward to advancing discussions with the agency.
About Primary Biliary Cholangitis
Primary biliary cholangitis (PBC) is a rare, progressive, and chronic autoimmune disease that affects the bile ducts in the liver and is most prevalent (approximately 1 in 10,000) in women over the age of 40. PBC causes bile acid to build up in the liver, resulting in inflammation and scarring (fibrosis), which, if left untreated, can lead to cirrhosis, a liver transplant, or death.
About Ocaliva® (obeticholic acid)
OCALIVA, a farnesoid X receptor (FXR) agonist, is indicated for the treatment of adult patients with primary biliary cholangitis (PBC)
- without cirrhosis or
- with compensated cirrhosis who do not have evidence of portal hypertension, either in combination with ursodeoxycholic acid (UDCA) with an inadequate response to UDCA or as monotherapy in patients unable to tolerate UDCA.
This indication is approved under accelerated approval based on a reduction in alkaline phosphatase (ALP). An improvement in survival or disease-related symptoms has not been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
About Intercept Pharmaceuticals
Intercept is a biopharmaceutical company and a wholly owned subsidiary of Alfasigma S.p.A. focused on the development and commercialization of novel therapeutics to treat rare and serious liver diseases, including primary biliary cholangitis (PBC) and severe alcohol-associated hepatitis (sAH). Intercept owns the commercial rights to Ocaliva in the U.S. market.
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