

A Remarkable
Turning Point
Impacting the lives of those with rare liver disease is more than a mission—it’s our calling. At Mirum, our work is in service of closing treatment gaps to open a world of possibilities for patients and their families.
Patients and families play a crucial role in our clinical trial designs and approaches. We work with them to help identify what matters most as our investigational treatments are evaluated. This includes the development of surveys, pruritus (itch) severity scales, and e-diaries that help create a better understanding of the burdens of rare liver diseases.



Development pipeline
LIVMARLI® (maralixibat) Oral Solution |
Preclinical | Phase 1 | Phase 2b/Phase 3 | Approved |
---|---|---|---|---|
Alagille Syndrome (ALGS) |
FDA Approved MAA submitted in Europe | |||
Progressive Familial Intrahepatic Cholestasis (PFIC) |
MARCH | |||
Biliary Atresia (BA) | EMBARK |
VOLIXIBAT | ||||
---|---|---|---|---|
Primary Sclerosing Cholangitis (PSC) |
VISTAS | |||
Intrahepatic Cholestasis of Pregnancy (ICP) |
OHANA | |||
Primary Biliary Cholangitis (PBC) |
VANTAGE |
MAA: Marketing Authorization Application
Oral Solution
FDA Approved | APPROVED |
MAA submitted in Europe |
Oral Solution
Cholestasis (PFIC)
MARCH Phase 2b/Phase 3 |
Oral Solution
EMBARK Phase 2b/Phase 3 | APPROVED |
Cholangitis (PSC)
VISTAS Phase 2b/Phase 3 | APPROVED |
of Pregnancy (ICP)
OHANA Phase 2b/Phase 3 | APPROVED |
Cholangitis (PBC)
VANTAGE Phase 2b/Phase 3 | APPROVED |
MAA: Marketing Authorization Application
Learn more about our investigational therapies
LIVMARLI® (maralixibat)
Volixibat
Our clinical
trials
At Mirum, working together with the rare liver disease community is paramount.
Patients and their families play an integral role in helping us evaluate investigational
therapies through clinical trials and obtain the evidence we need for regulatory
approval in the U.S. and beyond.
For more information about Mirum’s clinical trials, please contact:
- Healthcare providers: clinicaltrials@mirumpharma.com
- Patients and families: medinfo@mirumpharma.com (U.S. & International [non-Europe]);
medinfoEU@mirumpharma.com (Europe)
Clinical trials for LIVMARLI® (maralixibat)
Clinical trials for volixibat
Expanded access program
References
- Gonzales E, Sturm E, Stormon E, et al. Durability of treatment effect with long-term maralixibat in children with Alagille syndrome. Oral presentation at: American Association for the Study of Liver Diseases Annual Meeting (The Liver Meeting); November 8-12, 2019; Boston, MA.
- Mirum Pharmaceuticals Announces Breakthrough Therapy Designation for Maralixibat for the Treatment of Pruritus Associated with Alagille Syndrome. Mirum Pharmaceuticals, Inc. Accessed June 2, 2021. https://ir.mirumpharma.com/news-releases/news-release-details/mirum-pharmaceuticals-announces-breakthrough-therapy-designation
- Mirum Pharmaceuticals Completes Successful Pre-NDA Meeting with FDA for Maralixibat. Mirum Pharmaceuticals, Inc. Accessed June 2, 2021.
- Mirum Pharmaceuticals Announces Completion of Rolling NDA Submission for Maralixibat in Alagille Syndrome. Mirum Pharmaceuticals, Inc. Accessed June 2, 2021. https://ir.mirumpharma.com/news-releases/news-release-details/mirum-pharmaceuticals-announces-completion-rolling-nda
- Thompson R. Serum bile acid control in long-term maralixibat-treated patients is associated with native liver survival in children with progressive familial intrahepatic cholestasis due to bile salt export pump deficiency. Presented at: EASL 2020; August 2020. Accessed April 29, 2021.
- Key CC, McKibben A, Chien E, et al. A Phase 1 dose-ranging study assessing fecal bile acid excretion by volixibat, an apical sodium‑dependent bile acid transporter inhibitor, and coadministration with loperamide.