United States: Intercept Pharmaceuticals’ Ocaliva for the treatment of liver disease has not received a full backing from the U.S. FDA though currently benefits from an accelerated approval.
Ocaliva’s Current Approval Status
Ocaliva stays available as a treatment for PBC, a rare liver disease, thanks to the FDA accelerated approval program. This pathway lets drugs be approved from the preliminary study; however, it needs other clinical trials to establish efficacy, according to Reuters.
Problems That lie Ahead for Ocaliva’s Confirmatory Trials
Actual clinical trials of Ocaliva were stopped prematurely because of the challenges of finding suitable patients with PBC a disease that affects less than 200,000 new patients every year in the U.S. This has created doubts on how it will hold in the market and even threatened with withdrawal if future trials will not demonstrate its effectiveness.
Analyst Reactions
Nonetheless, according to Julia Wattacheril from Columbian University, Ocaliva’s future is now dependent on whether Intercept Pharmaceuticals can acquire more data from other trials. However some issues arise how much the company is willing to bring resource to this cause.
Increasing Competition in the Market
Ocaliva, along with Ursodiol, has been one of the few approved treatments for PBC. However, the market now includes alternatives such as Gilead’s Livdelzi, creating additional competition for Intercept’s drug, as reported by Reuters.
Global Regulatory Setbacks
Earlier this year, Ocaliva faced a setback in Europe, where the European Medicines Agency (EMA) revoked its conditional authorization due to insufficient patient benefits, further complicating the drug’s future in global markets.