A Remarkable
Turning Point
Impacting the lives 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 |
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LIVMARLI® |
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Alagille Syndrome |
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LIVMARLI® |
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Progressive Familial Intrahepatic |
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VOLIXIBAT |
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Primary Sclerosing |
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VOLIXIBAT |
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Primary Biliary |
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CHENODAL® |
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Cerebrotendinous |
1 Approved in the below regions:
- U.S. – In cholestatic pruritus in patients with ALGS three months of age and older
- E.U. – In cholestatic pruritus in patients with ALGS two months of age and older
- Canada – In cholestatic prutitus 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.
2 Approved in the below regions:
- U.S. – In cholestatic pruritus in patients with PFIC 12 months of age and older
- E.U. – For the treatment of PFIC in patients three months of age and older
3 Blinded Interim analysis for VISTAS study in PSC announced on June 17, 2024; study continues as planned
4 Interim data from the Phase 2b VANTAGE clinical trial examining the safety and efficacy of volixibat for the treatment of PBC was announced on June 17, 2024
5 Chenodal (CDCA) is not indicated for CTX but has received a medical necessity determination in the U.S. by the FDA for CTX. Data from the Phase 3 RESTORE clinical trial examining the safety and efficacy of Chenodal for the treatment of CTX was announced on October 2, 2023.
LIVMARLI®
(maralixibat) Oral Solution
Alagille Syndrome
(ALGS)
Approved |
LIVMARLI®
(maralixibat) Oral Solution
Progressive Familial Intrahepatic
Cholestasis (PFIC)
Phase 2b/Phase 3 |
VOLIXIBAT
Primary Sclerosing
Cholangitis (PSC)
Phase 2b/Phase 3 |
VOLIXIBAT
Primary Biliary
Cholangitis (PBC)
Phase 2b/Phase 3 |
CHENODAL®
(chenodiol)
Cerebrotendinous
xanthomatosis (CTX)
Phase 2b/Phase 3 |
1 Approved in the below regions:
- U.S. – In cholestatic pruritus in patients with ALGS three months of age and older
- E.U. – In cholestatic pruritus in patients with ALGS two months of age and older
- Canada – In cholestatic prutitus 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.
2 Approved in the below regions:
- U.S. – In cholestatic pruritus in patients with PFIC 12 months of age and older
- E.U. – For the treatment of PFIC in patients three months of age and older
3 Blinded Interim analysis for VISTAS study in PSC announced on June 17, 2024; study continues as planned
4 Interim data from the Phase 2b VANTAGE clinical trial examining the safety and efficacy of volixibat for the treatment of PBC was announced on June 17, 2024
5 Chenodal (CDCA) is not indicated for CTX but has received a medical necessity determination in the U.S. by the FDA for CTX. Data from the Phase 3 RESTORE clinical trial examining the safety and efficacy of Chenodal for the treatment of CTX was announced on October 2, 2023.