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 |
---|---|---|---|---|
LIVMARLI® |
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Alagille Syndrome |
||||
LIVMARLI® |
||||
Progressive Familial Intrahepatic |
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VOLIXIBAT |
||||
Primary Sclerosing |
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VOLIXIBAT |
||||
Primary Biliary |
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CHENODAL® |
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Cerebrotendinous |
* 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.
**** In cholestatic pruritus in patients with PFIC five years of age and older.
***** 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 |
* 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.
**** In cholestatic pruritus in patients with PFIC five years of age and older.
***** 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.