Maralixibat clinical trials
Maralixibat is named as an intervention in 4 current clinical trials in our USA, Germany, France, Spain, Italy, UK, Poland, Canada, Brazil, Netherlands, Belgium, Portugal and Japan registers. 3 are recruiting patients now. The most common phase is Observational. The sponsor with the most trials is Mirum Pharmaceuticals, Inc (3). The most-studied condition is Alagille Syndrome.
Where Maralixibat is approved
All regulators →Of the three regulators tracked here, the first to approve Maralixibat was the US, on 29 Sep 2021. It is approved in 3 of the 3 regulators tracked here.
- 1 EU orphan designation, most recently for treatment of biliary atresia (27 Jul 2020, positive)
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
Maralixibat patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Maralixibat, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 10 listed patents; the last listed expires 5 Oct 2043 (US 12296050).
- Exclusivity ODE-490: Orphan drug exclusivity: treatment of cholestatic pruritas in patients 12 months of age to less than 5 years of age with progressive famili…, until 24 Jul 2031.
- Exclusivity ODE-471: Orphan drug exclusivity: treatment of cholestatic pruritus in patients 5 years of age and older with progressive familial intrahepatic chol…, until 13 Mar 2031.
- Exclusivity ODE*: FDA has not recognized orphan-drug exclusivity (ode) for this drug, but it contains the same active moiety or moieties as another drug(s) t…, until 13 Mar 2031.
- Exclusivity ODE-429: Orphan drug exclusivity: treatment of cholestatic pruritus in patients 3 months of age to less than 1 year of age with alagille syndrome (a…, until 13 Mar 2030.
- Exclusivity ODE*: FDA has not recognized orphan-drug exclusivity (ode) for this drug, but it contains the same active moiety or moieties as another drug(s) t…, until 13 Mar 2030.
- Exclusivity ODE-379: Orphan drug exclusivity: for treatment of cholestatic pruritus in patients with alagille syndrome (algs) 1 year of age and older, until 29 Sep 2028.
- 2 further exclusivities are listed.
- Livmarli for treatment of Alagille syndrome: until 12 Dec 2034 (includes 24 months' paediatric extension). Union Register
- Livmarli for treatment of progressive familial intrahepatic cholestasis: until 1 Jul 2036 (includes 24 months' paediatric extension). Union Register
Reproduced from the FDA Orange Book and Purple Book (checked 8 Nov 2026) and the European Commission's Union Register of medicinal products (CC BY 4.0; the EU expiry date is calculated exactly as the Commission's register displays it). Listed patents and dates are as submitted to or published by the regulator and may not reflect litigation, invalidation, settlements, lapsed fees or later extensions. This is not legal advice or a freedom-to-operate opinion, and it does not say when a generic or biosimilar will launch.
What health systems spend on Maralixibat
All medicines →From NHSBSA Prescription Cost Analysis (Open Government Licence v3.0) and CMS State Drug Utilization Data. Both are gross amounts: neither system publishes its discounts or rebates per drug. Products combining several substances are not counted here.
How Maralixibat works
All drug targets →- Ileal bile acid transporter inhibitor InhibitorTarget: SLC10A2 solute carrier family 10 member 2✓ In ChEMBL and the Guide to Pharmacology
- Alagille syndromePhase 3
- Progressive familial intrahepatic cholestasisPhase 3
- Constipation disorderPhase 2/3
- Cholestatic liver diseasePhase 2
- Intrahepatic cholestasis of pregnancyPhase 2
- Biliary atresiaPhase 2
- Liver disorderPhase 2
- Sclerosing cholangitisPhase 2
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL363392, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 11708, CC BY-SA 4.0. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
Patient leaflets for Maralixibat
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 6 products on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EUEuropean Union: LivmarliEMA product information: package leaflet and summary of product characteristics in every EU language.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
Reference identifiers for Maralixibat
- ChEMBL
- CHEMBL363392
- DrugBank
- DB16226
- PubChem CID
- 9831643
- CAS number
- 716313-53-0
- FDA UNII
- UYB6UOF69L
- Wikidata
- Q27291331
Codes that identify this medicine in other databases, from Wikidata (CC0), matched by its ChEMBL ID. The ATC code is the WHO classification of what the medicine is used for.
Trials by status
Trials by phase
Clinical trials of Maralixibat
4 trialsRegisters list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.
Maralixibat across our data
Ireland · EU · USThe same active substance in our other registers, matched by its exact name. Each entry links to the register record it comes from.
Licensed in Ireland (HPRA)
6 single-substance products containing Maralixibat on the HPRA medicines list.
- Livmarli 10 mg tabletsMirum Pharmaceuticals International B.V. · EU/1/22/1704/003
- Livmarli 15 mg tabletsMirum Pharmaceuticals International B.V. · EU/1/22/1704/004
- Livmarli 19 mg/mL oral solutionMirum Pharmaceuticals International B.V. · EU/1/22/1704/002
- Livmarli 20 mg tabletsMirum Pharmaceuticals International B.V. · EU/1/22/1704/005
- Livmarli 30 mg tabletsMirum Pharmaceuticals International B.V. · EU/1/22/1704/006
Supply shortages
No entry for Maralixibat in the EMA, FDA or German BfArM shortage lists we track.
Conditions studied with Maralixibat
5 conditionsSponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Mirum Pharmaceuticals, Inc | 3 |
| Takeda | 1 |
By country register
A trial with sites in more than one country appears in each of those registers.
Related compounds
Questions about Maralixibat trials
How many clinical trials are testing Maralixibat?
4 current trials in our USA, Germany, France, Spain, Italy, UK, Poland, Canada, Brazil, Netherlands, Belgium, Portugal and Japan registers name Maralixibat (also registered as Formerly LUM001) as a drug or biological intervention, as of the registry sync on 10 November 2026. Completed and withdrawn studies are not counted.
Are Maralixibat trials recruiting patients?
Yes. 3 of the 4 trials are recruiting: 1 with sites in USA, 1 with sites in Germany, 1 with sites in France, 1 with sites in Spain, 1 with sites in Italy, 1 with sites in Netherlands, 1 with sites in Belgium, 1 with sites in Portugal and 1 with sites in Japan. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Maralixibat clinical trials?
Mirum Pharmaceuticals, Inc (3) and Takeda (1). The number in brackets is how many trials each organisation leads.
Is Maralixibat licensed in Ireland?
Yes. The HPRA medicines list includes 6 single-substance products containing Maralixibat, for example Livmarli 10 mg tablets (Mirum Pharmaceuticals International B.V.). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Maralixibat shortage?
Not in the lists we track: Maralixibat has no entry in the EMA, FDA or German BfArM shortage registers as of their latest sync.
Where does this information come from?
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Maralixibat works or whether it is effective, and it is not medical advice.
How this page is built
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Maralixibat works or whether it is effective. Each trial links to its own record so you can check it yourself.
- ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, intervention names and synonyms, phase, overall status, lead sponsor and conditions for every trial on this page
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Maralixibat (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 10 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.