Emerging Treatment Options in PBC

Opinion
Video

Sonal Kumar, MD, MPH, and David Victor III, MD, provide an overview of the emerging treatments in the primary biliary cholangitis landscape, highlighting PPAR agonists and combination OCA and bezafibrate.

Kris Kowdley, MD, FACP, FACG, AGAF, FAASLD: There are a lot of new things, like new medications in development. Seladelpar is in a phase 3 trial that’s close to enrollment. Elafibranor is in a phase 3 trial that’s close to enrollment. These are PPAR-Δ and PPAR-⍺-Δ dual agonists. Saroglitazar, which is an PPAR-⍺-γ dual agonist, is also in clinical trials. There’s a fixed-dose combination study that’s ongoing with obeticholic acid and bezafibrate. Sonal, what’s your impression about these trials? Have you been involved? What’s the future in terms of these new therapies? Are you excited about them?

Sonal Kumar, MD, MPH: We’ve been involved in some of these trials. It’s exciting…. We’re using different mechanisms of action to attack this disease. The more we learn about it and the more drugs we have in our toolbox, it’s better for patient outcomes, especially in patients who are symptomatic. If they’re not a candidate for things like obeticholic acid, PPARs are a great option for them. I’m excited to see the combo with obeticholic acid and bezafibrate. I know it’s still in phase 2 studies, though.

Kris Kowdley, MD, FACP, FACG, AGAF, FAASLD: Thanks. Does anybody else participating in these trials want to comment on how our treatment landscape may be changing in the next few years?

Steven Flamm, MD, FAASLD, FACG: We’re participating in some of the trials. The preliminary data from some of them are very impressive. Keep in mind that these studies are in patients who are failing or intolerant to ursodiol. There aren’t new studies, to my knowledge, with these emerging agents looking like primary therapies for primary biliary cholangitis [PBC]. I’d be most excited about that because it wouldn’t be a surprise if these products help patients who are the most difficult, the ones who are failing ursodiol or not having an optimal response. If they’re safe, there’s no reason to believe they wouldn’t also help ursodiol in treatment of PBC as a primary indication. We’re not looking at that yet, but I’m excited for this particular patient population, these new products.

David Victor III, MD: I’m excited that there’s some excitement around PBC. I hope this unearths patients, in primary care and GI [gastrointestinal] practices, who’ve been languishing on ursodiol who we may be able to do more for. The best day of my life would be if we never did another liver transplant. I hope that, with these new agents, we’re moving that way for PBC. It’s an exciting time for a disease state that everyone knew how to treat, but we’re bringing back options and opportunities to treat them better. It’s a great time for PBC and the patients. They’ve been treated fairly, but now we have more.

Kris Kowdley, MD, FACP, FACG, AGAF, FAASLD: That’s an excellent point, Steve, about step-up therapy. Steve and I are probably the only 2 individuals old enough to remember the old step-up vs step-down PPI [proton pump inhibitor] debates when we were fellows and doing gastroenterology: start with antacids and then go up to a PPI.

Steven Flamm, MD, FAASLD, FACG: Thanks a lot, Kris.

Kris Kowdley, MD, FACP, FACG, AGAF, FAASLD: Well, you’re the only individual I can say this to.

Steven Flamm, MD, FAASLD, FACG: That’s true.

Kris Kowdley, MD, FACP, FACG, AGAF, FAASLD: Everybody else is the next generation. It’s interesting to think that you start with a more effective therapy, you could potentially withdraw ursodiol. This is a real issue for patients. The cost is not a trivial matter. It seems to be less of a complaint now, but a lot of my patients, especially those who are on Medicare, will have problems affording UDCA [ursodeoxycholic acid]. I encourage sponsors and individuals doing clinical trials to say you could redraw or have a monotherapy study and see what happens with these therapies.

Transcript edited for clarity

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