Intercept's Ocaliva granted accelerated approval by FDA for primary biliary cholangitis

Intercept Pharmaceuticals announced that the FDA awarded accelerated approval to Ocaliva (obeticholic acid) for the treatment of adults with primary biliary cholangitis (PBC). Specifically, the farnesoid X receptor agonist was authorised for use in combination with ursodeoxycholic acid in patients with an inadequate response to ursodeoxycholic acid or as monotherapy in adults who could not tolerate ursodeoxycholic acid. 

Approval of the therapy was supported by safety and efficacy data from the Phase III POISE trial. In the study, the results of which were initially reported in 2014, Ocaliva met its primary endpoint of a reduction in serum alkaline phosphatase levels to below a threshold of 1.67 times the upper limit of normal. Specifically, the endpoint was achieved in 46 percent of patients in the titration group, compared to 10 percent of placebo-treated patients. 

Intercept cautioned that an improvement in survival or disease-related symptoms has not been established for the therapy, adding that continued approval for this indication could be contingent upon the verification and description of clinical benefit in confirmatory trials. The company has initiated patient enrolment in a study of the effects of Ocaliva on rates of liver failure and death, with the study projected to be completed by 2023. 

In April, an FDA advisory panel unanimously backed approval of Ocaliva for the treatment of adults with PBC, which followed a staff review that identified no major concerns regarding the drug's use for this indication (for related analysis, see ViewPoints: Intercept’s Ocaliva receives a not-so-ringing endorsement from FDA panel). The FDA had previously delayed a decision regarding the approval of the drug and asked Intercept to provide additional clinical data. 

Meanwhile, Intercept is additionally developing Ocaliva for the treatment of non-alcoholic steatohepatitis (NASH), for which the therapy was granted breakthrough therapy status by the FDA in 2015. Earlier this month, the company announced its intention to conduct a late-stage study of Ocaliva for the treatment of patients with NASH and liver fibrosis.  In 2014, the drugmaker halted the mid-stage FLINT trial of Ocaliva in NASH early after the therapy met the main goal of the study. Intercept later provided a positive trial update to address concerns regarding a safety signal related to lipid dysregulation (for additional analysis, read KOL Views: Assessing recent developments in NASH). 

Earlier this month, RBC Capital Markets analyst Michael Yee estimated that the global market for NASH could be worth between $5 billion and $10 billion. Meanwhile, analysts have forecast sales of $1.6 billion for Ocaliva by 2020. Intercept indicated that the therapy is expected to be launched within seven to 10 days. 

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