Maralixibat chloride, sold under the brand name Livmarli, is a

medication A medication (also called medicament, medicine, pharmaceutical drug, medicinal drug or simply drug) is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy (pharmacotherapy) is an important part of the medical field and re ...

used to treat cholestatic pruritus in people with

Alagille syndrome Alagille syndrome is a genetic disorder that affects primarily the liver and the heart. Problems associated with the disorder generally become evident in infancy or early childhood. The disorder is inherited in an autosomal dominant pattern, and t ...

. Maralixibat chloride is an

ileal bile acid transporter Ileal sodium/bile acid cotransporter, also known as apical sodium–bile acid transporter (ASBT) and ileal bile acid transporter (IBAT), is a bile acid:sodium symporter protein that in humans is encoded by the ''SLC10A2'' gene. ASBT/IBAT is mos ...

(IBAT) inhibitor. Maralixibat was approved for medical use in the United States in September 2021.

History

Maralixibat was granted

orphan drug An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of ...

designations in 2013, and in 2020.

Society and culture

Legal status

On 13 October 2022, the

Committee for Medicinal Products for Human Use The Committee for Medicinal Products for Human Use (CHMP), formerly known as Committee for Proprietary Medicinal Products (CPMP), is the European Medicines Agency's committee responsible for elaborating the agency's opinions on all issues regarding ...

(CHMP) of the

European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of medicinal products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or Euro ...

(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. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.

Names

Maralixibat chloride is the international nonproprietary name (INN).

References

External links

* * * Drugs acting on the gastrointestinal system and metabolism Orphan drugs Heterocyclic compounds with 2 rings Sulfur heterocycles Nitrogen heterocycles Quaternary ammonium compounds Tertiary amines {{gastrointestinal-drug-stub