Mirum Pharmaceuticals has two approved indications for its IBAT inhibitor Livmarli (maralixibat) — Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis — and is advancing volixibat, another IBAT inhibitor, in Phase 2b/3 for Primary Sclerosing Cholangitis and Primary Biliary Cholangitis. Competitors are active in all four spaces, with odevixibat (Albireo/Ipsen) and TAK-625 (Takeda) in late-stage trials for ALGS/PFIC, and linerixibat (GSK) and obeticholic acid (Intercept) among others in PSC/PBC. Recent FDA documents confirm Livmarli’s approved label expansions and tablet formulation, while volixibat has no dedicated FDA filings yet. ---
Improvements in pruritus, serum bile acids, hepatic fibrosis, and portal hypertension in two Alagille syndrome patients treated with maralixibat — maralixibat (treatment cohort/case series)
Improvements in pruritus and serum bile acids, alongside potential regression of liver fibrosis and portal hypertension in two Alagille syndrome patients — maralixibat (treatment cohort/case series)
Improvements in pruritus, serum bile acids, liver fibrosis, and portal hypertension in two Alagille syndrome patients treated with maralixibat — maralixibat (treatment cohort/case series)
4FDA documents
NDA Risk Assessment and Risk Mitigation Review — Livmarli (maralixibat): evaluates whether a REMS is necessary for Livmarli for cholestatic pruritus in ALGS.
NDA review — LIVMARLI (maralixibat): multi-discipline and labeling reviews for the NDA for ALGS.
Corrected NDA Approval Letter — Livmarli (maralixibat): corrected approval letter for tablets to treat cholestatic pruritus in ALGS or PFIC.
Label — LIVMARLI (maralixibat): indicated for cholestatic pruritus in ALGS or PFIC.
Supplement approval letter — Livmarli (maralixibat): sNDA approval to expand prescribing information to include ALGS patients aged 3 months and older.
Approval Letter — Livmarli (maralixibat): approval of oral solution for ALGS patients 1 year and older.
Prescribing Information (Label) — LIVMARLI (maralixibat) oral solution: indicated for ALGS and PFIC.
NDA/BLA Multi-Disciplinary Review and Evaluation — Maralixibat (Livmarli): evaluation of new immediate-release tablet for ALGS and PFIC.
Takeaway Livmarli is already approved for ALGS, but two Phase 3 competitors (odevixibat, TAK-625) are in the pipeline; Mirum’s advantage is its approved label and multiple FDA filings supporting pediatric age expansions.
Biochemical improvement after resuming ursodeoxycholic acid (UDCA) therapy in a PFIC3 patient with ABCB4 mutation — ursodeoxycholic acid (UDCA) (treatment cohort/case series)
Improvement in liver function following UDCA therapy in a PFIC3 patient — ursodeoxycholic acid (UDCA) (treatment cohort/case series)
Moderate biochemical improvement after UDCA therapy in a PFIC3 patient with ABCB4 mutation — ursodeoxycholic acid (UDCA) therapy (treatment cohort/case series)
4FDA documents
NDA/BLA Multi-Disciplinary Review and Evaluation — Livmarli (maralixibat chloride): evaluation of new tablet formulation for ALGS or PFIC.
Prescribing Information — LIVMARLI (maralixibat): indicated for cholestatic pruritus in ALGS (≥3 months) and PFIC (≥12 months).
Approval Letter — Livmarli (maralixibat): approval of tablets for ALGS (≥3 months) or PFIC (≥12 months, ≥25 kg).
Label — LIVMARLI (maralixibat): indicated for ALGS and PFIC.
Prescribing Information / Label — LIVMARLI (maralixibat) oral solution: indicated for ALGS (≥3 months) and PFIC (≥12 months).
Prescribing Information (Label) — LIVMARLI (maralixibat): indicated for ALGS (≥3 months) and PFIC (≥12 months).
Prescribing Information (Label) — LIVMARLI (maralixibat): indicated for ALGS (≥3 months) and PFIC (≥5 years).
Takeaway Livmarli is approved for PFIC, but competitors odevixibat and TAK-625 are in Phase 3; Mirum’s label covers a broad age range, but the PFIC competitor landscape is crowded with multiple Phase 3 IBAT inhibitors.
Maralixibat improved cholestatic pruritus, reduced serum bile acids, and was associated with regression of liver fibrosis and portal hypertension in two Alagille syndrome patients — maralixibat (treatment cohort/case series)
Maralixibat improved cholestatic pruritus, reduced serum bile acids, and showed potential benefits in regressing liver fibrosis and improving portal hypertension in two Alagille syndrome patients — maralixibat (treatment cohort/case series)
4FDA documents
NDA/BLA Multi-Disciplinary Review and Evaluation — Livmarli (maralixibat chloride): evaluation of tablet formulation for ALGS and PFIC.
Label — LIVMARLI (maralixibat): indicated for ALGS (≥3 months) and PFIC (≥12 months).
Labeling — LIVMARLI (maralixibat): same indication.
Label — LIVMARLI (maralixibat): indicated for ALGS or PFIC.
Prescribing Information (Label) — LIVMARLI (maralixibat): indicated for ALGS (≥3 months) and PFIC (≥12 months).
Prescribing Information (Label) — LIVMARLI (maralixibat) oral solution: indicated for ALGS (≥3 months) and PFIC (≥12 months).
Corrected NDA Approval Letter — Livmarli (maralixibat) tablets: for ALGS or PFIC.
Risk Assessment and Risk Mitigation Review — Livmarli (maralixibat): REMS not required.
Approval Letter — Livmarli (maralixibat): corrected approval for ALGS or PFIC.
NDA review — Livmarli (maralixibat): REMS not required.
Label — LIVMARLI (maralixibat): indicated for ALGS (≥3 months) and PFIC (≥12 months).
FDA reviews — LIVMARLI (maralixibat): multidisciplinary reviews for ALGS.
Prescribing Information (Label) — LIVMARLI (maralixibat): indicated for ALGS and PFIC.
Prescribing Information (Label) — LIVMARLI (maralixibat): indicated for ALGS (≥3 months) and PFIC (≥12 months).
NDA/BLA Multi-Disciplinary Review and Evaluation — Livmarli (maralixibat chloride): evaluation of tablet formulation.
Label — LIVMARLI (maralixibat) oral solution: indicated for ALGS (≥3 months) and PFIC (≥12 months).
Takeaway This indication aggregates ALGS/PFIC pruritus trials; Mirum’s Livmarli is already approved for both, but competitors like odevixibat and TAK-625 are in Phase 3 for the same patient populations, creating direct head-to-head competition.
Review of pathogenesis of PSC and evaluation of current off-label treatments and emerging therapeutic candidates — Ursodeoxycholic acid (UDCA), norucholic acid (NCA), obeticholic acid (OCA), and other emerging therapies (treatment review/meta-analysis)
Protocol for a randomized, placebo-controlled phase 2 trial of oral vancomycin in PSC with or without IBD — oral vancomycin (clinical trial)
Takeaway Volixibat is in early-stage development for PSC, facing a crowded field of Phase 2/3 competitors including linerixibat (GSK, Phase 3) and obeticholic acid (Intercept). No volixibat-specific FDA documents were found, indicating a nascent regulatory path.
NDA Approval Letter — Lynavoy (linerixibat): approved for cholestatic pruritus in PBC.
Administrative and Correspondence Documents / Meeting Minutes — seladelpar (MBX-8025): FDA meeting minutes.
Takeaway Volixibat is in early-stage development for PBC, competing against multiple Phase 3 agents (linerixibat, seladelpar) and the approved obeticholic acid. No volixibat-specific FDA documents exist, highlighting a significant gap versus competitors with established regulatory interactions.