I attended the 2025 International PBC Summit in April of this year. The event was sponsored by several pharmaceutical companies directly involved with the treatment of patients with primary biliary cholangitis (PBC). On day three of the Summit, patients and patient advocates had the opportunity to sit down with each pharmaceutical company. The pharmaceutical reps presented recent information related to clinical trials of their PBC treatments, and answered questions from patients. I really learned a lot that day, and I want to share it.
What I learned about treatment developments
First let’s start with Ocaliva (obeticholic acid), a second-line treatment for PBC. Ocaliva is made by Intercept in the U.S. and Advanz Pharma outside of the U.S. Last year the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) decided against full approval of this medication. There were issues with previous clinical trial standards preventing full approval. I learned at the Summit that Ocaliva is still available for patients and had not been taken off of the market, which is what I had previously thought. The medicine is still available under accelerated approval. Many patients use Ocaliva with great success.
Next let’s go to Gilead. They make Livdelzi (seladelpar) which is a second-line treatment for PBC. Livdelzi was previously sold by CymaBay Therapeutics, but Gilead bought CymaBay. Livdelzi has been shown during the Phase 3 clinical trials to really help with the itch and fatigue patients with PBC experience. Livdelzi was given accelerated approval in 2024 for treatment of PBC. Because it is new, Livdelzi can be expensive so I asked Gilead how patients can receive financial assistance. They pointed me to their LIVDELZI Co-Pay Coupon Program.
Ipsen received accelerated approval in 2024 as well, for Iqirvo (elafibranor), which is a second-line treatment for PBC. Phase 3 clinical trials showed a big improvement with patient itch. At the Summit, it was mentioned that some trial participants saw their alkaline phosphatase, or ALP, decrease within four weeks of starting the medication. I thought that was pretty incredible, especially since ALP is one of the biggest lab markers for PBC. I also asked Ipsen for information on financial assistance they provide, because I’ve seen many patients say they cannot afford it. I learned they have the IPSEN CARES patient support program.
Now that we’ve covered all of the second-line therapies, let’s talk about PBC itch. There are two different medications in the works for pruritus. GSK talked with us about Linerixibat; they recently applied for New Drug Approval with the U.S. FDA. Everything GSK told us left me with a lot of hope for patients who experience the dreaded itch. Their reps said the GLISTEN Phase 3 trials showed a big improvement on the itch as well as itch-related sleep issues. Hopefully we will be receiving medication approval in the near future!
Mirum also has been working on anti-itch medication called Volixibat. Mirum presented data from their Phase 2B VANTAGE trial which also seems very promising. Mirum told us that 70% of patients in the trial showed evidence of 50% or more reduction in bile salts. (Increased levels of bile salts is what causes the itch with PBC.) I also learned that originally, Mirum developed Livmarli to treat itching in two different pediatric liver diseases and they found great success with the medication. Volixibat is getting ready for Phase 3 trials.
And finally, I saved the medication I am most excited about for last. Umecrine Cognition has been working on Golexanolone, which is used for fatigue and cognitive impairment specifically with PBC. I first learned about this clinical trial when I was at the 2024 International PBC Summit. I couldn’t wait to hear updates on Golexanolone this year, because fatigue and brain fog are my personal worst symptoms of PBC. Golexanolone is currently getting ready for a Phase 2 trial in PBC, however, they have already completed a Phase 2 trial and are getting ready for a Phase 3 trial as a treatment for hepatic encephalopathy. The representative talked about how this medication helps to balance out the allopregnanolone levels in the brain. High levels of allopregnanolone are the cause of fatigue, brain fog, cognitive impairment and other harmful effects that can cause dysfunction. With hepatic encephalopathy, they saw patients report better sleep, cognitive function and better levels of fatigue. They are hoping that the Phase 2 studies in PBC will show the same thing. One thing about this medication that I found really interesting is that Umecrine is hopeful this medication will be able to be used for all different disease processes that cause fatigue and cognitive impairment. Umecrine told us that currently all trials have been in Europe, but they are hoping to expand to the United States in the future. This is a medication I would love to be part of the clinical trials for. I am so hopeful that Golexanolone will be able to help all different patients that experience this very limiting symptom. I will most certainly be keeping my eyes peeled for Golexanolone in the future!
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