Ocaliva plus bezafibrate clears FDA orphan drug hurdle for primary biliary cholangitis
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The FDA granted Intercept Pharmaceuticals orphan drug designation for the fixed-dose combination of obeticholic acid and bezafibrate intended for treatment of patients with primary biliary cholangitis.
Obeticholic acid (Ocaliva, Intercept Pharmaceuticals) — a farnesoid X receptor agonist — was originally FDA approved for primary biliary cholangitis (PBC) in 2016 in combination with ursodeoxycholic acid. The new regimen pairs obeticholic acid with bezafibrate, a peroxisome proliferator-activated receptor agonist.
“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,” M. Michelle Berrey, MD, MPH, president of research & development and chief medical officer of Intercept, said in the release. “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 FDA based its decision on Intercept’s two ongoing phase 2 trials that assessed different therapeutic doses for the combination of OCA and bezafibrate. The company stated that it anticipates completing the interim analysis of both trials in 2023, with research being presented at the upcoming European Association for the Study of the Liver Congress, being held from June 21-24, 2023, in Vienna, Austria.
Intercept expects to use the planned interim analyses from the phase 2 studies, as well as phase 1 and preclinical data as basis for a potential end-of-phase 2 meeting with the FDA.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
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