The PSC drug pipeline
There is no approved medicine for PSC yet. These are the drugs being tested in Phase 2 and Phase 3 PSC trials, built from ClinicalTrials.gov records, plus the latest public news on the two furthest along.
Investigational drugs are not approved and may never be. Results summarized here come from company and conference reports and are not advice to seek any treatment. Talk to your hepatologist.
Closest to a decision
Hand-checked against the linked sources on 2026-10-17.
Norucholic acid (nor-UDCA, NCA)
Dr. Falk Pharma · A modified bile acid, related to UDCA (ursodiol).
Where it stands: Phase 3 completed (NUC-5); under regulatory review in the EU and Australia
- Listed now Study with Norucholic Acid Tablets in Patients with Primary Sclerosing Cholangitis (PSC) (Phase 3, Not yet recruiting, NCT06886360) Source: ClinicalTrials.gov record
- 2026-09-15 The EMA's CHMP agenda for 14-17 September 2026 lists a pre-authorisation oral explanation for norucholic acid (orphan, EMEA/H/C/006515) for PSC in adults, scheduled 15 September 2026. The September meeting highlights did not list an opinion for it. Source: EMA CHMP draft agenda, 14-17 September 2026
- 2026-05 At the EASL Congress 2026, a NUC-5 subgroup analysis reported the effect was seen with or without background UDCA, and appeared larger in patients not taking UDCA (23.4% vs 0% without UDCA; 12.7% vs 5.1% with UDCA). Source: European Medical Journal, EASL 2026 congress review
- 2025-09 Australia's TGA lists YORLIVI (norucholic acid) for PSC in adults as a new medicine under evaluation (accepted September 2025). Source: Therapeutic Goods Administration (Australia)
- 2025-05-07 Dr. Falk Pharma announced that the Phase 3 NUC-5 trial (NCT03872921, 301 patients) met its primary endpoint at 96 weeks: 15.1% of patients on norucholic acid vs 4.2% on placebo reached the combined endpoint (partial normalization of alkaline phosphatase and no worsening of disease stage on biopsy). Safety event rates were similar between groups. Source: Dr. Falk Pharma press release
- 2021-06-24 FDA orphan drug designation for treatment of PSC (designated; not FDA approved). Source: FDA Orphan Drug Designations database
Volixibat
Mirum Pharmaceuticals · An oral IBAT inhibitor (it blocks bile acids from being reabsorbed in the gut), studied here for itch (cholestatic pruritus) in PSC.
Where it stands: Phase 2b (VISTAS) met primary endpoint; NDA submission now targeted for first half of 2027
- 2026-08-05 Mirum reported FDA granted Breakthrough Therapy and Orphan Drug designations for volixibat in cholestatic pruritus due to PSC. At the pre-NDA meeting the FDA recommended a Phase 3 study; Mirum plans further FDA discussions and now targets a potential NDA submission in the first half of 2027. Source: Mirum Q2 2026 results (SEC exhibit 99.1)
- 2026-05-30 Full VISTAS results presented at EASL 2026: 56% of volixibat patients had at least a 2-point itch reduction vs 26% on placebo. The most common side effects were diarrhea and abdominal pain; liver test elevations were seen more often with volixibat. Source: Mirum press release (EASL 2026) and published abstract
- 2026-05-04 Mirum announced the Phase 2b VISTAS study (NCT04663308) met its primary endpoint for itch in PSC: a 2.72-point reduction from baseline on the 0-10 Adult ItchRO scale, 1.64 points better than placebo (p<0.0001). Source: Mirum Pharmaceuticals press release
All late-stage PSC drug trials
Phase 2 and Phase 3 PSC drug trials on ClinicalTrials.gov that are recruiting, active, recently completed or recently stopped (records updated since 2022). Grouped by drug; placebo arms left out. Data from 2026-10-17.
| Drug | Furthest phase | Trials |
|---|
| Cilofexor | Phase 3 | |
|---|
| Elafibranor | Phase 3 | |
|---|
| Norucholic acid (nor-UDCA, NCA) | Phase 3 | - NCT06886360 · Phase 3 · Not yet recruiting · Dr. Falk Pharma GmbH
- NCT03872921 · Phase 3 · UNKNOWN · Dr. Falk Pharma GmbH
|
|---|
| Simvastatin | Phase 3 | - NCT04133792 · Phase 3 · Active, not recruiting · Annika Bergquist
|
|---|
| Vancomycin | Phase 3 | - NCT07341282 · Phase 2 · Recruiting · McMaster University
- NCT07646223 · Phase 2 · Not yet recruiting · Tampere University Hospital
- NCT03710122 · Phase 2 / Phase 3 · Terminated · Elizabeth Carey
- NCT05876182 · Phase 2 · Recruiting · University of Milano Bicocca
|
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| BRS201 | Phase 2 | - NCT05835505 · Phase 2 · Recruiting · Brigham and Women's Hospital
|
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| CS0159 | Phase 2 | - NCT05896137 · Phase 2 · Active, not recruiting · Cascade Pharmaceuticals, Inc
|
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| DUR-928 | Phase 2 | |
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| EP547 | Phase 2 | - NCT05525520 · Phase 2 · Completed · Escient Pharmaceuticals, Inc
|
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| Fecal microbiota transplantation | Phase 2 | - NCT07477782 · Phase 2 · Not yet recruiting · Assistance Publique - Hôpitaux de Paris
|
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| Fenofibrate | Phase 2 | |
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| HK-660S | Phase 2 | - NCT06975150 · Phase 2 · Not yet recruiting · CuromeBiosciences
- NCT05866809 · Phase 2 · Completed · CuromeBiosciences
|
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| HTD1801 | Phase 2 | - NCT03333928 · Phase 2 · Completed · HighTide Therapeutics (Hong Kong) Limited
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| Hymecromone | Phase 2 | |
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| LB-P8 | Phase 2 | - NCT06699121 · Phase 2 · Recruiting · LISCure Biosciences
|
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| Nemolizumab-ilto | Phase 2 | - NCT07843420 · Phase 2 · Not yet recruiting · Mayo Clinic
|
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| PLN-74809 | Phase 2 | - NCT04480840 · Phase 2 · Completed · Pliant Therapeutics, Inc.
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| Ritivixibat | Phase 2 | - NCT05642468 · Phase 2 · Terminated · Albireo, an Ipsen Company
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| Seladelpar | Phase 2 | |
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| Sulfasalazine | Phase 2 | - NCT03561584 · Phase 2 · Completed · Brigham and Women's Hospital
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| Upadacitinib | Phase 2 | - NCT07678645 · Phase 2 · Not yet recruiting · RenJi Hospital
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| Vidofludimus calcium | Phase 2 | |
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| Volixibat | Phase 2 | - NCT04663308 · Phase 2 · Active, not recruiting · Mirum Pharmaceuticals, Inc.
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