Pharma Focus America

Intercept Pharmaceuticals Receives FDA Orphan Drug Designation for the Fixed-Dose Combination of OCA and Bezafibrate

Wednesday, May 17, 2023

Intercept Pharmaceuticals, Inc. (Nasdaq: ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for the fixed-dose combination of obeticholic acid (OCA) and bezafibrate, a peroxisome proliferator-activated receptor (PPAR) agonist, for the potential treatment of individuals with primary biliary cholangitis (PBC).

“We are pleased that the FDA has granted orphan drug designation for the fixed-dose combination of OCA-bezafibrate, an important component of our long-term strategy and ongoing commitment to people living with PBC,” said M. Michelle Berrey, MD, MPH, President of Research & Development and Chief Medical Officer of Intercept. “This designation represents a milestone in the development of the OCA-bezafibrate fixed-dose combination, which we believe provides the potential to establish best-in-class clinical benefits and further improve the treatment of PBC.”

Intercept has two ongoing Phase 2 studies (747-213 / NCT04594694, 747-214 / NCT05239468) that are designed to explore a range of therapeutic doses for the combination of OCA and bezafibrate. The company expects to complete planned interim analyses from both ongoing Phase 2 studies in 2023, with the first data being presented at the 2023 European Association for the Study of the Liver (EASL) Congress, which will be held from June 21-24, 2023, in Vienna, Austria. The planned interim analyses from these Phase 2 studies, in addition to Phase 1 and preclinical data, will serve as the basis of a potential end-of-phase 2 meeting with the FDA.

FDA's Office of Orphan Drug Products grants orphan status to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, including market exclusivity upon regulatory approval, exemption of FDA application fees, and tax credits for qualified clinical trials.

About the Investigational OCA-Bezafibrate Fixed-Dose Combination
Intercept is investigating a fixed-dose combination of OCA and bezafibrate for the potential treatment of individuals with PBC. The OCA-bezafibrate combination is investigational; safety and efficacy have not been established. OCA, a farnesoid X receptor (FXR) agonist, is marketed by Intercept as Ocaliva in the United States for the treatment of PBC. Bezafibrate, a pan-peroxisome proliferator-activated receptor (pan-PPAR) agonist, is not approved in the United States for any indication.

FXR and PPAR are distinct pathways that each play a role in PBC. Simultaneously targeting both pathways may offer the greatest potential to impact bile acid synthesis, metabolism, and clearance that underlie cholestatic liver diseases. Published studies establish a clinical proof-of-concept that suggests that the combination of OCA and bezafibrate may provide additive clinical efficacy and tolerability benefits in the treatment of PBC.

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