Intercept Pharmaceuticals has received orphan drug designation from US Food and Drug Administration (FDA) for the fixed-dose combination of obeticholic acid (OCA) and bezafibrate to treat individuals with primary biliary cholangitis (PBC).
OCA is a farnesoid X receptor (FXR) agonist, which is marketed as Ocaliva by Intercept in the US to treat PBC.
Bezafibrate is a pan-peroxisome proliferator-activated receptor (pan-PPAR) agonist, which is not approved in the US for any indication.
A rare, progressive, and chronic autoimmune disease, primary biliary cholangitis (PBC) impacts the bile ducts in the liver and is most prevalent in women – of approximately 1 in 10,000 – over the age of 40.
Intercept president of research & development and chief medical officer M. Michelle Berrey said: “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.
“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.”
The firm has two ongoing Phase 2 studies (747-213 / NCT04594694, 747-214 / NCT05239468) to explore an array of therapeutic doses for the combination of OCA and bezafibrate.
It expects to finish planned interim analyses from both the ongoing Phase 2 studies this year, with the first data being presented at the 2023 European Association for the Study of the Liver (EASL) Congress, which will be held from in Vienna, Austria from 21-24 June.
The interim analyses from these Phase 2 trials, in addition to Phase 1 and preclinical data, will form the basis of a potential end-of-phase 2 meeting with the regulatory agency.