New Potential Primary Biliary Cholangitis Treatment Granted Breakthrough Therapy Designation

Researchers at GENFIT are excited about promising results from studies investigating a new potential therapy for PBC and NASH, two rare liver diseases.

Primary Biliary Cholangitis

Primary biliary cholangitis (PBC) is a rare liver disease. The disease causes damage to the bile ducts located in the liver, which impairs the body’s ability to get rid of toxins. It can lead to pruritus, cirrhosis, and ultimately, liver failure. Unfortunately, half of PBC patients do not respond to current treatment options or have side effects that force them to stop taking the medication.

One of the common treatments many patients have an inadequate response to is called ursodeoxycholic acid (UDCA).

Researchers at GENFIT believe they may have found another option for both PBC and NASH. It’s called elafibranor.

Nonalcoholic Steatohepatitis

Nonalcoholic steatohepatitis (NASH) is another rare liver disease that causes fat to accumulate in the body. It can cause inflammation, cirrhosis, liver insufficiency, and even liver cancer.


Elafibranor is a dual PPAR alpha/delta agonist which is taken orally, once each day. It has been in development for both PBC and NASH. Researchers believe it could be beneficial for patients who have had an inadequate response to other treatment.

The drug is currently in a Phase 3 trial for NASH, which was initiated following positive results from a Phase 2 investigation.

A Phase 3 clinical trial for PBC should be initiated this year, also following a positive Phase 2 trial. It was the results from this Phase 2 trial which convinced the FDA to grant elafibranor Breakthrough Therapy Designation for PBC.

Phase 2 Trial for PBC

This was a 12 week trial investigating elafibranor as a treatment for adults with PBC who had an inadequate response to the typical UDCA treatment.

The results from this trial were presented at the European Association for the Study of the Liver’s annual International Liver Congress.

The goal of this trial was to determine the safety and the efficacy of elafibranor for this patient population. Participants were all non-cirrhotic adults with PBC who had not had an adequate response to UDCA. Two different doses of the treatment were evaluated, and both were found to be generally well-tolerated.

Positive results included a decrease in ALP levels, improvements in GGT, total cholesterol, triglycerides, LDL, IgM, fibrinogen, haptoglobin, and CRP markers. These results were consistent with the results from the Phase 2 NASH trial.

GENFIT is excited about the new designation provided to elafibranor for PBC thanks to these trial results because it will help them to accelerate development of the treatment. Additionally, it confirms their belief that this drug could serve this population of patients who currently have a high unmet need.

You can read more about the development of elafibranor in these two rare liver diseases here.

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