Maralixibat Chloride

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Cat Number

API228113664

CAS Number

228113-66-4

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Product Detail Description Scientific Support Customer Q&A

CAS Number

228113-66-4

Storage

Store at 2-8°C

Synonyms

LUM-001; UNII-V78M04F0XC; Livmarli; V78M04F0XC; SHP 625

Molecular Formula

C40H56ClN3O4S

Molecular Weight

710.4

Smiles

CCCCC1(CS(=O)(=O)C2=C(C=C(C=C2)N(C)C)[C@H]([C@H]1O)C3=CC=C(C=C3)OCC4=CC=C(C=C4)C[N+]56CCN(CC5)CC6)CCCC.[Cl-]

Appearance

White to light yellow solid

General Description

Maralixibat chloride is a small-molecule inhibitor of the apical sodium-dependent bile acid transporter (ASBT), also known as the ileal bile acid transporter (IBAT). It acts locally in the gastrointestinal tract with minimal systemic absorption.

Mechanism of Action

Maralixibat selectively inhibits the ileal bile acid transporter (IBAT) located on the apical surface of enterocytes in the terminal ileum, preventing the reuptake of bile acids from the intestinal lumen into the portal circulation. This blockade interrupts the enterohepatic circulation of bile acids, leading to increased fecal excretion of bile salts and a consequent reduction in systemic and hepatic serum bile acid levels.

Application

Maralixibat is indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). Reducing serum bile acids ameliorates intractable itching associated with these rare pediatric liver diseases, and improvements in xanthoma severity have also been observed.

In an open‑label proof‑of‑concept study, the ileal bile acid transporter inhibitor maralixibat (≤10 mg/d for 14 weeks) was given to 27 adults with primary sclerosing cholangitis. Serum bile acid levels decreased by 16.7% overall and by 40% in those with elevated baseline levels. In participants with moderate‑to‑severe pruritus at baseline, itch scores improved significantly (70% reduction in those with ItchRO daily average ≥3). Most adverse events were mild or moderate gastrointestinal symptoms (diarrhea 51.9%). Maralixibat reduces serum bile acids and improves pruritus in PSC, warranting further study.

Fig. 1 Pruritus outcomes. (Bowlus CL, et al., 2023)

References

Bowlus CL, et al. Safety, tolerability, and efficacy of maralixibat in adults with primary sclerosing cholangitis: Open-label pilot study. Hepatol Commun. 2023;7(6):e0153.

Does Maralixibat Chloride require refrigerated storage as a bile acid transport inhibitor?

Yes, it must be stored at 2-8°C. The compound is thermally labile; at room temperature, hydrolysis and aggregation may occur.

Is Maralixibat Chloride sensitive to light and moisture?

Yes, it is both photosensitive and hygroscopic. Store in original, tightly sealed, light-resistant containers with desiccant under refrigeration.

What is the stability of Maralixibat Chloride in oral suspension formulations?

Reconstituted suspensions have limited stability (typically 30 days under refrigeration).

How is the impurity maralixibat free base (dechlorination product) monitored?

This degradation product is specifically quantified using a stability-indicating HPLC method, ensuring it remains within ICH limits.