Maralixibat chloride

Maralixibat chloride
Clinical data
Trade namesLivmarli
Other namesLUM001
AHFS/Drugs.comMonograph
MedlinePlusa621056
License data
Routes of
administration		By mouth
Drug classIleal bile acid transporter (IBAT) inhibitor
ATC code
Legal status
Legal status
Identifiers
  • 1-[4-({4-[(4R,5R)-3,3-Dibutyl-7-(dimethylamino)-4-hydroxy-1,1-dioxido-2,3,4,5-tetrahydro-1-benzothiepin-5-yl]phenoxy}methyl)benzyl]-4-aza-1-azoniabicyclo[2.2.2]octane
CAS Number
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
CompTox Dashboard (EPA)
Chemical and physical data
FormulaC40H56ClN3O4S
Molar mass710.42 g·mol−1
3D model (JSmol)
  • CCCCC1(CCCC)CS(=O)(=O)C2=CC=C(C=C2[C@H]([C@H]1O)C1=CC=C(OCC2=CC=C(C[N+]34CCN(CC3)CC4)C=C2)C=C1)N(C)C
  • InChI=1S/C40H56N3O4S/c1-5-7-19-40(20-8-6-2)30-48(45,46)37-18-15-34(41(3)4)27-36(37)38(39(40)44)33-13-16-35(17-14-33)47-29-32-11-9-31(10-12-32)28-43-24-21-42(22-25-43)23-26-43/h9-18,27,38-39,44H,5-8,19-26,28-30H2,1-4H3/q+1/t38-,39-/m1/s1
  • Key:STPKWKPURVSAJF-LJEWAXOPSA-N

Maralixibat chloride, sold under the brand name Livmarli, is a medication used to treat cholestatic pruritus in people with Alagille syndrome.[5][6] Maralixibat chloride is an ileal bile acid transporter (IBAT) inhibitor.[5]

The most common side effects include diarrhea and abdominal pain (belly ache).[6]

Maralixibat chloride was approved for medical use in the United States in September 2021,[5][7][8][9] and in the European Union in December 2022.[6]

Medical uses

Maralixibat chloride is indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome.[5][6]

History

The U.S. Food and Drug Administration (FDA) granted the application for maralixibat chloride orphan drug designations in 2013,[10][11] and in 2020.[12][13]

Society and culture

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).[14] The applicant for this medicinal product is Mirum Pharmaceuticals International B.V.[14] Maralixibat chloride was approved for medical use in the European Union in December 2022.[6][15]

Names

Maralixibat chloride is the international nonproprietary name (INN).[16]

References

  1. ^ "Details for: Livmarli". Health Canada. 11 August 2023. Retrieved 3 March 2024.
  2. ^ "Summary Basis of Decision (SBD) for Livmarli". Health Canada. 1 September 2012. Retrieved 11 December 2023.
  3. ^ "Notice: Multiple additions to the Prescription Drug List (PDL) [2023-10-26]". Health Canada. 26 October 2023. Retrieved 3 January 2024.
  4. ^ "Regulatory Decision Summary for Livmarli". Drug and Health Products Portal. 21 July 2023. Retrieved 2 April 2024.
  5. ^ a b c d e "Livmarli- maralixibat chloride solution". DailyMed. Archived from the original on 1 November 2021. Retrieved 31 October 2021.
  6. ^ a b c d e f "Livmarli EPAR". European Medicines Agency (EMA). 12 October 2022. Archived from the original on 14 March 2023. Retrieved 4 March 2023. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  7. ^ "Maralixibat: FDA-Approved Drugs". U.S. Food and Drug Administration (FDA). Archived from the original on 30 September 2021. Retrieved 29 September 2021.
  8. ^ "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.
  9. ^ Shirley M (January 2022). "Maralixibat: First Approval". Drugs. 82 (1): 71–76. doi:10.1007/s40265-021-01649-0. PMC 8748361. PMID 34813049.
  10. ^ "Maralixibat Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 4 September 2013. Archived from the original on 30 September 2021. Retrieved 29 September 2021.
  11. ^ "Maralixibat Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 4 September 2013. Archived from the original on 30 September 2021. Retrieved 29 September 2021.
  12. ^ "Maralixibat Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 21 October 2020. Archived from the original on 30 September 2021. Retrieved 29 September 2021.
  13. ^ Advancing Health Through Innovation: New Drug Therapy Approvals 2021 (PDF). U.S. Food and Drug Administration (FDA) (Report). 13 May 2022. Archived from the original on 6 December 2022. Retrieved 22 January 2023. Public Domain This article incorporates text from this source, which is in the public domain.
  14. ^ a b "Livmarli: Pending EC decision". European Medicines Agency. 14 October 2022. Archived from the original on 25 October 2022. Retrieved 15 October 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  15. ^ "Livmarli Product information". Union Register of medicinal products. Archived from the original on 16 December 2022. Retrieved 3 March 2023.
  16. ^ World Health Organization (2016). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 75". WHO Drug Information. 30 (1). hdl:10665/331046.
  • 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