FDA grants priority review and sets PDUFA date for obeticholic acid (OCA) for primary biliary cirrhosis- Intercept Pharma

Intercept Pharmaceuticals, Inc. announced the FDA has accepted for review the company's New Drug Application and granted Priority Review for obeticholic acid (OCA) for the treatment of primary biliary cirrhosis (PBC).

OCA is being developed to treat PBC patients with an inadequate therapeutic response to, or who are unable to tolerate, ursodeoxycholic acid (UDCA), the only drug currently approved to treat PBC. The FDA has set a target date of 29 February 2016 to take action under the Prescription Drug User Fee Act (PDUFA).

A priority review designation means FDA's goal is to take action on the marketing application within six months of acceptance as compared to 10 months under standard review. OCA was previously granted Fast Track designation by FDA. A drug development programme with Fast Track designation is afforded greater access to FDA for the purpose of expediting the drug's development, review and potential approval.

Comment: OCA brought elevated levels of pruritus, and also 10 serious cardiovascular events and two deaths in the OCA arm of the FLINT study. Since Primary Biliary Cirrhosis is an autoimmune disease for which patients would be expected to be on lifelong treatment, the pruritus problem could be of concern. However, Intercept's CEO Dr Mark Pruzanski stated that starting patients on a lower dose of 5mg and titrating up to 10mg "sacrificed very little in efficacy but resulted in a very clear improvement in tolerability".