Maralixibat chloride explained

Tradename:Livmarli
Dailymedid:Maralixibat chloride
Routes Of Administration:By mouth
Class:Ileal bile acid transporter (IBAT) inhibitor
Atc Prefix:A05
Atc Suffix:AX04
Legal Ca:Rx-only
Legal Ca Comment:[1] [2] [3] [4]
Legal Us:Rx-only
Legal Us Comment:[5]
Legal Eu:Rx-only
Index2 Label:as salt
Cas Number:716313-53-0
Cas Number2:228113-66-4
Drugbank:DB16226
Drugbank2:DBSALT003196
Chemspiderid:8007375
Chemspiderid2:8007374
Unii:UYB6UOF69L
Unii2:V78M04F0XC
Kegg:D10951
Kegg2:D10952
Chembl:363392
Chembl2:17879
Synonyms:LUM001
Iupac Name:1-[4-({4-[(4''R'',5''R'')-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
C:40
H:56
N:3
O:4
S:1
Cl:1
Smiles: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
Stdinchi: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
Stdinchikey:STPKWKPURVSAJF-LJEWAXOPSA-N

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

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

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

Medical uses

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

History

The U.S. Food and Drug Administration (FDA) granted the application for maralixibat chloride orphan drug designations in 2013,[9] [10] and in 2020.[11] [12]

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). The applicant for this medicinal product is Mirum Pharmaceuticals International B.V.[13] Maralixibat chloride was approved for medical use in the European Union in December 2022.[14] [15]

Names

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

Notes and References

  1. Web site: Details for: Livmarli . . 11 August 2023 . 3 March 2024.
  2. Web site: Summary Basis of Decision (SBD) for Livmarli . . 1 September 2012 . 11 December 2023.
  3. Web site: Notice: Multiple additions to the Prescription Drug List (PDL) [2023-10-26] ]. . 26 October 2023 . 3 January 2024.
  4. Web site: Regulatory Decision Summary for Livmarli . Drug and Health Products Portal . 21 July 2023 . 2 April 2024.
  5. Web site: Livmarli- maralixibat chloride solution . DailyMed . 31 October 2021 . 1 November 2021 . https://web.archive.org/web/20211101040959/https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=64000394-1ef6-4e76-8ba8-11f25ba1b167 . live .
  6. Web site: Maralixibat: FDA-Approved Drugs . U.S. Food and Drug Administration (FDA) . 29 September 2021 . 30 September 2021 . https://web.archive.org/web/20210930060552/https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=214662 . live .
  7. 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 . Business Wire . 29 September 2021 . 29 September 2021 . 20 October 2022 . https://web.archive.org/web/20221020182744/https://www.businesswire.com/news/home/20210929005810/en/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 . live .
  8. Shirley M . Maralixibat: First Approval . Drugs . 82 . 1 . 71–76 . January 2022 . 34813049 . 8748361 . 10.1007/s40265-021-01649-0 . free . doi .
  9. Web site: Maralixibat Orphan Drug Designations and Approvals . U.S. Food and Drug Administration (FDA) . 4 September 2013 . 29 September 2021 . 30 September 2021 . https://web.archive.org/web/20210930062624/https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=406013 . live .
  10. Web site: Maralixibat Orphan Drug Designations and Approvals . U.S. Food and Drug Administration (FDA) . 4 September 2013 . 29 September 2021 . 30 September 2021 . https://web.archive.org/web/20210930062623/https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=401513 . live .
  11. Web site: Maralixibat Orphan Drug Designations and Approvals . U.S. Food and Drug Administration (FDA) . 21 October 2020 . 29 September 2021 . 30 September 2021 . https://web.archive.org/web/20210930062623/https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=705519 . live .
  12. Advancing Health Through Innovation: New Drug Therapy Approvals 2021 . U.S. Food and Drug Administration (FDA) . 13 May 2022 . PDF . 22 January 2023 . 6 December 2022 . https://web.archive.org/web/20221206210020/https://www.fda.gov/media/155227/download . live .
  13. Web site: Livmarli: Pending EC decision . European Medicines Agency . 14 October 2022 . 15 October 2022 . 25 October 2022 . https://web.archive.org/web/20221025204936/https://www.ema.europa.eu/en/medicines/human/summaries-opinion/livmarli . dead . Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  14. Web site: Livmarli EPAR . European Medicines Agency (EMA) . 12 October 2022 . 4 March 2023 . 14 March 2023 . https://web.archive.org/web/20230314125424/https://www.ema.europa.eu/en/medicines/human/EPAR/livmarli . live . Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  15. Web site: Livmarli Product information . Union Register of medicinal products . 3 March 2023 . 16 December 2022 . https://web.archive.org/web/20221216114513/https://ec.europa.eu/health/documents/community-register/html/h1704.htm . live .
  16. ((World Health Organization)) . 2016 . International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 75 . WHO Drug Information . 30 . 1 . 10665/331046 . free . World Health Organization .

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