Dear Registry Community,

This spring marks a truly exciting moment for PSC. New treatments for PSC, once considered a distant possibility, are closer than ever before.

In the U.S., the successful Phase 2b trial for volixibat to treat itch will hopefully lead to a New Drug Application for FDA review later this year. In Europe, norucholic acid was submitted for European marketing approval last August following a successful Phase 3 trial. The new global Phase 3 trial for elafibranor in PSC is expected to start enrolling within the next few months. Other potential treatments continue to move forward, with 28 active or pending trials for PSC globally.

On behalf of the Registry Team, thank you to the thousands of people living with PSC who have participated in clinical trials. That’s a big number for a rare disease, but we can only get there one patient at a time. Pharmaceutical companies have told us that learning of the number of active Registry participants has helped encourage them to pursue PSC treatments. Your participation in the Registry is directly accelerating research.

- Brian Thorsen, Director, PSC Partners Patient Registry

Results from Phase 2b Trial for Volixibat to Treat Itch in PSC

Results are now available from the VISTAS study for volixibat to treat pruritus (itch) in people living with PSC. The study achieved its primary endpoint of improvement in itch for those receiving volixibat. Mirum Pharmaceuticals plans to meet with the FDA for a New Drug Application for volixibat to be hopefully submitted in the second half of 2026.

This marks the first successful late-stage clinical trial in the U.S. for new potential treatments for PSC, a major milestone for the community.

Now Available for ALL: Gallbladder Removal in PSC

A new survey focusing on the impact of gallbladder removal surgery on the course and outcomes of PSC is now available in the Registry.

To effectively compare outcomes, this survey is open to ALL Registry participants. Caregivers can complete the survey on behalf of their loved ones with PSC.

2025 Registry Impact Report

The Registry's new annual Impact Report details major accomplishments, projects, and the five publications made possible by Registry participants in the last year. In addition, there are key statistics for all participants which represents one of the largest single datasets on people living with PSC.

Learning From Registry Data

This chart was created using the most recent Clinical Survey responses from 2,465 Registry participants as of April 2026. According to our data, about 32% of PSCers have had their gallbladder removed. Among those without a liver transplant, the gallbladder rate is 28%. This lower rate can be partially explained by the need for gallbladder removal during transplant, but the Gallbladder Removal in PSC survey examines the association between the two procedures in greater detail.

In Focus: Patient-Reported Symptoms

The April 2026 issue of The Beacon focuses on the effects of common PSC symptoms on those experiencing them; the lack of patient-reported symptom measurement tools; and how PSC Partners’ Symptom Assessment Project (SAP) is addressing this need. Your participation in the project through surveys and focus groups is making a difference!

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