

A Remarkable
Turning Point
Impacting the livers of those with rare 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 diseases.



Development pipeline
LIVMARLI® (maralixibat) Oral Solution |
Preclinical | Phase 1 | Phase 2b/Phase 3 | Approved |
---|---|---|---|---|
Alagille Syndrome (ALGS) |
FDA Approved* EC-Authorized** Health Canada Approved*** | |||
Progressive Familial Intrahepatic Cholestasis (PFIC) |
MARCH | |||
Biliary Atresia (BA) | EMBARK |
VOLIXIBAT | ||||
---|---|---|---|---|
Primary Sclerosing Cholangitis (PSC) |
VISTAS | |||
Primary Biliary Cholangitis (PBC) |
VANTAGE |
CHENODAL® (chenodiol) |
||||
---|---|---|---|---|
Cerebrotendinous xanthomatosis (CTX) |
RESTORE**** |
* In cholestatic pruritus in patients with ALGS three months of age and older
** In cholestatic pruritus in patients with ALGS two months of age and older
*** In cholestatic pruritus in patients with ALGS
- Pediatrics (12 months to 18 years): Based on the data submitted and reviewed by Health Canada, the safety and efficacy of LIVMARLI in these pediatric patients have been established. Therefore, Health Canada has authorized an indication for pediatric use.
- Pediatrics (<12 months): The safety and efficacy of LIVMARLI in these pediatric patients have not been established.
****Chenodal (CDCA) is not indicated for CTX but has received a medical necessity determination in the U.S. by the FDA for CTX. The Phase 3 RESTORE clinical trial is currently underway to examine the safety and efficacy of Chenodal® (CDCA) for the treatment of CTX.
Topline data for Phase 3 MARCH study announced on October 24, 2022.
Oral Solution
FDA Approved* | APPROVED |
EC Approved** |
Health Canada Approved*** |
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 |
Cholangitis (PBC)
VANTAGE Phase 2b/Phase 3 | APPROVED |
RESTORE**** Phase 2b/Phase 3 | APPROVED |
* In cholestatic pruritus in patients with ALGS three months of age and older
** In cholestatic pruritus in patients with ALGS two months of age and older
*** In cholestatic pruritus in patients with ALGS
- Pediatrics (12 months to 18 years): Based on the data submitted and reviewed by Health Canada, the safety and efficacy of LIVMARLI in these pediatric patients have been established. Therefore, Health Canada has authorized an indication for pediatric use.
- Pediatrics (<12 months): The safety and efficacy of LIVMARLI in these pediatric patients have not been established.
****Chenodal (CDCA) is not indicated for CTX but has received a medical necessity determination in the U.S. by the FDA for CTX. The Phase 3 RESTORE clinical trial is currently underway to examine the safety and efficacy of Chenodal® (CDCA) for the treatment of CTX.
Topline data for Phase 3 MARCH study announced on October 24, 2022.
Learn more about our investigational therapies
LIVMARLI® (maralixibat)
Volixibat
Chenodal® (chenodiol)
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
Clinical trial for Chenodal® (chenodiol)
Expanded Access/Compassionate Use
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.