Mirum Pharmaceuticals
Mirum Pharmaceuticals works on 7 rare diseases tracked on Trial Friend, including Alagille Syndrome, Cerebrotendinous Xanthomatosis, Fibrodysplasia Ossificans Progressiva and 4 more, with 7 active clinical trials (4 recruiting) and 4 FDA-approved rare disease drugs.
Mirum Pharmaceuticals is a Foster City, California company focused on rare liver diseases, and it has quietly assembled one of the more productive commercial portfolios in the space. Its flagship, Livmarli (maralixibat), blocks bile acid recycling in the gut and is approved for cholestatic itching in two devastating pediatric liver conditions: Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC). For children whose relentless itch once led to liver transplant evaluations, an oral daily medicine changed the calculus.
The portfolio extends further: Cholbam (cholic acid) treats bile acid synthesis disorders, and Ctexli (chenodiol) became the first FDA-approved treatment specifically for cerebrotendinous xanthomatosis (CTX) in adults, a genetic disease long treated only off-label. On September 25, 2026 the FDA approved Atebrioz (zilurgisertib), licensed from Incyte, for fibrodysplasia ossificans progressiva (FOP) in people 12 and older. In the pipeline, volixibat applies the same bile-acid mechanism to adult cholestatic diseases. Its Phase 2b VISTAS study in primary sclerosing cholangitis met its primary endpoint on itch in May 2026, and Mirum is targeting an FDA filing in the first half of 2027. The VANTAGE study in primary biliary cholangitis has completed enrollment, with results expected in the first quarter of 2027. Brelovitug, an antibody for chronic hepatitis delta, met its primary endpoint in the Phase 3 AZURE-1 study on September 28, 2026, and MRM-3379 is in a Phase 2 study in Fragile X syndrome.
Mirum's pattern is consistent: take unglamorous, well-understood bile-acid biology and turn it into approved medicines for diseases most companies overlook.
Mirum Pharmaceuticals Drug Pipeline
Mirum Pharmaceuticals has 7 active clinical trials across 4 development stages, with 4 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Mirum Pharmaceuticals Clinical Trials (7)
Active and recruiting clinical trials sponsored by Mirum Pharmaceuticals, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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Mirum Pharmaceuticals FDA-Approved Drugs (4)
Medications developed or marketed by Mirum Pharmaceuticals that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| ZILURGISERTIB | ATEBRIOZ | Sep 25, 2026 | |
| CHOLIC ACID Bile Acid [EPC] | CHOLBAM oral | — | Mar 17, 2015 |
| CHENODIOL Bile Acid [EPC] | CTEXLI oral | Feb 21, 2025 | |
| MARALIXIBAT CHLORIDE | Livmarli oral | Sep 29, 2021 |
Mirum Pharmaceuticals Trial Locations
Mirum Pharmaceuticals clinical trials are running at 310 sites in 20 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (7)
Diseases targeted by Mirum Pharmaceuticals's clinical trial and drug development programs
Alagille syndrome is a rare autosomal dominant disorder affecting the liver, heart, skeleton, face, and eyes. The condition results from mutations in genes regulating the Notch signaling pathway, lead...
Cerebrotendinous xanthomatosis (CTX) is a rare inherited metabolic disorder caused by mutations in the CYP27A1 gene, which produces an enzyme essential for converting cholesterol into bile acids. The ...
Fibrodysplasia Ossificans Progressiva (FOP) is an extremely rare genetic disorder characterized by progressive heterotopic ossification, where soft tissues (muscles, tendons, ligaments) gradually tran...
Fragile X Syndrome is the most common inherited cause of intellectual disability and autism spectrum disorder. It results from mutations in the FMR1 gene that reduce production of fragile X mental ret...
Primary Biliary Cholangitis (PBC) is a chronic autoimmune liver disease in which the immune system mistakenly attacks the small bile ducts in the liver, leading to bile duct destruction and progressiv...
Primary Sclerosing Cholangitis (PSC) is a rare chronic liver disease characterized by inflammation and progressive scarring of the bile ducts, both inside and outside the liver. This leads to bile duc...
Patient Resources
Organizations and resources related to Mirum Pharmaceuticals's rare disease focus areas
Frequently Asked Questions About Mirum Pharmaceuticals
Common questions about Mirum Pharmaceuticals's rare disease programs, clinical trials, and treatments.