VIDEO: Recent approvals, developments in biliary diseases

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There are several drugs that have been both approved or about to be approved that have finished phase 3 trials. Several years ago, obeticholic acid, or also called Ocaliva, was approved by the FDA as a second-line therapy for patients with primary biliary cholangitis. That has led to an improvement in deaths and transplant-free survival of about 70%, according to one study.

At the moment, there’s a lot of interest in two other drugs that have just published or are in the process of publishing their phase 3 data, and that is the drug elafibranor and the drug seladelpar. These two drugs are agonists of the nuclear receptor, PPAR. These drugs have very similar properties. They both have met primary composite endpoint, which includes the reduction of the alkaline phosphatase below 1.67 and greater than 15% reduction in the total value of the alkaline phosphatase, and maintaining a normal bilirubin. So that’s the so-called composite endpoint that these trials use as their primary endpoint.

They both met that endpoint, although the percentage of cases that did so is slightly different. Elafibranor, I believe, was about 45%, and seladelpar was 60%. But if you compare the controls, which in the placebo group for seladelpar there was a 20% improvement, surprisingly, in the placebo group [and] if you subtract that from the 65, I think it is percent, you come down to 45. That’s very close to elafibranor. I don't see much difference between the drugs in terms of meeting their primary endpoint. Now, the secondary endpoint, one of the secondary endpoints was the normalization of the alkaline phosphatase.

The normalization was significantly different in the two drugs, it was I believe 25% for seladelpar and 15% for elafibranor. And then there was a difference in the response to itching, and the NRS score was significantly improved in the seladelpar trial, whereas it did not meet a statistical significance in the elafibranor trial. The elafibranor trial did have some improvement in itching as measured by other nonquantitative measurements. They both had about the same amount of side effects, which were minimal. But my guess is that the FDA will probably approve both drugs.