Fixed-dose combination OCA/Bezafibrate granted orphan drug designation by FDA
Intercept Pharmaceuticals is investigating a fixed-dose combination of OCA and bezafibrate for the potential treatment of individuals with PBC. Clinical trials are underway to establish safety and efficacy. OCA, a farnesoid X receptor (FXR) agonist is already licensed for the treatment of PBC in Canada. Bezafibrate, a pan-peroxisome proliferator-activated receptor (pan-PPAR) agonist, is used off-label for treatment of PBC.
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.