FDA OKs Intercept’s Orphan Drug
The US Food and Drug Administration (FDA) has granted accelerated approval for Intercept Pharmaceuticals’ Ocaliva (obeticholic acid) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as a single therapy in adults unable to tolerate UDCA.
PBC is a chronic disease that causes the small bile ducts in the liver to become inflamed, damaged and ultimately destroyed. This causes bile to remain in the liver, which damages the liver cells over time, and results in cirrhosis, or scarring of the liver. As cirrhosis progresses, and the amount of scar tissue in the liver increases, the liver loses its ability to function.
Ocaliva, given orally, binds to the farnesoid X receptor (FXR), a receptor found in the nucleus of cells in the liver and intestine. FXR is a key regulator of bile acid metabolic pathways. Ocaliva increases bile flow from the liver and suppresses bile acid production in the liver, thus reducing the exposure of the liver to toxic levels of bile acids.
Ocaliva was approved under the FDA’s accelerated approval program, which allows the approval of a drug to treat a serious or life-threatening disease based on clinical data showing the drug has an effect on a surrogate endpoint reasonably likely to predict clinical benefit to patients. This program provides earlier patient access to promising new drugs while the company conducts confirmatory clinical trials.
The FDA granted Ocaliva fast track designation, a process designed to facilitate the development, and expedite the review of drugs that are intended to treat serious conditions and that demonstrate the potential to address an unmet medical need. The FDA also granted Ocaliva an orphan drug designation.Orphan drug designationprovides incentives such as tax credits, user fee waivers and eligibility for orphan drug exclusivity to assist and encourage the development of drugs for rare diseases.