The Food and Drug Administration (FDA) has granted Fast Track designation to CNP-104 for the treatment of primary biliary cholangitis (PBC).

Primary biliary cholangitis (PBC) is a rare autoimmune liver disease characterized by immune-mediated destruction of the bile ducts. The E2 component of the pyruvate dehydrogenase complex (PDC-E2) is a common autoantigen in PBC. 

CNP-104 is an investigational biodegradable nanoparticle encapsulating PDC-E2. It utilizes the Company’s proprietary technology to combine disease specific pathogenic antigens with nanoparticles to mimic normal removal of dead or dying cells from the body.

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The Company is currently evaluating the efficacy, safety and tolerability of CNP-104 in adults with PBC in a phase 2 proof-of-concept study ( Identifier: NCT05104853). Patients will receive CNP-104 via intravenous infusion; primary outcome measures include frequency of adverse events and change in serum alkaline phosphatase levels.

“Based on data from another COUR autoimmune trial with a CNP platform product, our phase 2 PBC trial is designed to show that we can stop progression of PBC disease with improved liver function,” said COUR CEO John Puisis. “If successful in phase 2, COUR would seek breakthrough therapy designation in PBC given that current therapies treat only symptoms.”

The FDA’s Fast Track process allows for expedited review of drugs that are expected to fill an unmet need such as providing a therapy that may be better than currently available treatments.


  1. COUR Pharmaceuticals receives FDA Fast Track designation for CNP-104 for the treatment of primary biliary cholangitis. News release. COUR Pharmaceuticals. Accessed January 10, 2022. 
  2. Pipeline: CNP-104 (Primary Biliary Cholangitis). COUR Pharmaceuticals. Accessed January 10, 2022.
  3. US FDA accepts COUR Pharmaceuticals investigational New Drug Application (IND) for a Proof-of-concept study for the treatment of primary biliary cholangitis (PBC). News release. COUR Pharmaceuticals. October 4, 2021. Accessed January 10, 2022.

This article originally appeared on MPR