Chemistry:Maralixibat chloride
 | |
| Clinical data | |
|---|---|
| Trade names | Livmarli |
| Other names | LUM001 |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a621056 |
| License data | |
| Routes of administration | By mouth |
| Drug class | Ileal bile acid transporter (IBAT) inhibitor |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| |
| CAS Number |
|
| DrugBank |
|
| ChemSpider | |
| UNII |
|
| KEGG | |
| ChEMBL |
|
| Chemical and physical data | |
| Formula | C40H56ClN3O4S |
| Molar mass | 710.42 g·mol−1 |
| 3D model (JSmol) | |
| |
| |
Maralixibat chloride, sold under the brand name Livmarli, is a medication used to treat cholestatic pruritus in people with Alagille syndrome.[3][4] Maralixibat chloride is an ileal bile acid transporter (IBAT) inhibitor.[3]
The most common side effects include diarrhea and abdominal pain (belly ache).[4]
Maralixibat chloride was approved for medical use in the United States in September 2021,[3][5][6][7] and in the European Union in December 2022.[4]
Medical uses
Maralixibat chloride is indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome.[3][4]
History
The U.S. Food and Drug Administration (FDA) granted the application for maralixibat chloride orphan drug designations in 2013,[8][9] and in 2020.[10][11]
Society and culture
Legal status
In October 2022, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization under exceptional circumstances for the medicinal product Livmarli, intended for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS).[12] The applicant for this medicinal product is Mirum Pharmaceuticals International B.V.[12] Maralixibat chloride was approved for medical use in the European Union in December 2022.[4][13]
Names
Maralixibat chloride is the international nonproprietary name (INN).[14]
References
- ↑ "Summary Basis of Decision (SBD) for Livmarli". 1 September 2012. https://dhpp.hpfb-dgpsa.ca/review-documents/resource/SBD1698846935419.
- ↑ "Notice: Multiple additions to the Prescription Drug List (PDL) [2023-10-26"]. 26 October 2023. https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/prescription-drug-list/notices-changes/multiple-additions-2023-10-26.html.
- ↑ 3.0 3.1 3.2 3.3 3.4 "Livmarli- maralixibat chloride solution". https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=64000394-1ef6-4e76-8ba8-11f25ba1b167.
- ↑ 4.0 4.1 4.2 4.3 4.4 4.5 "Livmarli EPAR". 12 October 2022. https://www.ema.europa.eu/en/medicines/human/EPAR/livmarli. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ "Maralixibat: FDA-Approved Drugs". https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=214662.
- ↑ "U.S. FDA Approves Livmarli (maralixibat) as the First and Only Approved Medication for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome One Year of Age and Older". Mirum Pharmaceuticals (Press release). 29 September 2021. Archived from the original on 20 October 2022. Retrieved 29 September 2021 – via Business Wire.
- ↑ "Maralixibat: First Approval". Drugs 82 (1): 71–76. January 2022. doi:10.1007/s40265-021-01649-0. PMID 34813049.
- ↑ "Maralixibat Orphan Drug Designations and Approvals". 4 September 2013. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=406013.
- ↑ "Maralixibat Orphan Drug Designations and Approvals". 4 September 2013. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=401513.
- ↑ "Maralixibat Orphan Drug Designations and Approvals". 21 October 2020. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=705519.
- ↑ (PDF) Advancing Health Through Innovation: New Drug Therapy Approvals 2021 (Report). 13 May 2022. https://www.fda.gov/media/155227/download. Retrieved 22 January 2023.
This article incorporates text from this source, which is in the public domain.
- ↑ 12.0 12.1 "Livmarli: Pending EC decision". 14 October 2022. https://www.ema.europa.eu/en/medicines/human/summaries-opinion/livmarli. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ "Livmarli Product information". https://ec.europa.eu/health/documents/community-register/html/h1704.htm.
- ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 75". WHO Drug Information 30 (1). 2016.
External links
- Clinical trial number NCT02160782 for "Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS) (ICONIC)" at ClinicalTrials.gov
 |  |
