Related Experiment Video

Updated: Oct 12, 2025

A Simplified Stepwise Approach to Echo Guidance during Percutaneous Mitral Valve Repair
08:31

A Simplified Stepwise Approach to Echo Guidance during Percutaneous Mitral Valve Repair

Published on: October 16, 2021

4.0K

Maralixibat: First Approval

Matt Shirley1

  • 1Springer Nature, Mairangi Bay, Private Bag 65901, Auckland, 0754, New Zealand. dru@adis.com.

Drugs
|November 23, 2021
PubMed

Insights

Maralixibat, an ileal bile acid transporter inhibitor, is now approved in the USA for treating cholestatic pruritus in Alagille syndrome patients aged one and older. This marks a significant milestone for rare cholestatic liver disease treatment.

Area of Science:

  • Pharmacology
  • Hepatology
  • Drug Development

Background:

  • Rare cholestatic liver diseases, including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia, present significant unmet medical needs.
  • Ileal bile acid transporter (IBAT) inhibition is a therapeutic strategy for managing bile acid homeostasis in cholestatic conditions.

Purpose of the Study:

  • To summarize the key development milestones of maralixibat, an oral IBAT inhibitor.
  • To highlight the regulatory pathway leading to the first approval of maralixibat for Alagille syndrome.

Main Methods:

  • Review of preclinical and clinical development data for maralixibat.
  • Analysis of regulatory submissions and approvals for maralixibat in rare cholestatic liver diseases.

Main Results:

  • Maralixibat, a small-molecule IBAT inhibitor, has undergone extensive development for treating rare cholestatic liver diseases.
  • The drug received its first US approval on September 29, 2021, for cholestatic pruritus in ALGS patients aged one year and older.

Conclusions:

  • The US approval of maralixibat represents a significant advancement in the treatment of Alagille syndrome.
  • Continued development is underway for other pediatric cholestatic liver conditions, including PFIC and biliary atresia, and for younger ALGS patients.

More Related Videos

Scaled-Up Preparation of an Intermediate of Upatinib, ACT051-308:36

Scaled-Up Preparation of an Intermediate of Upatinib, ACT051-3

Published on: April 7, 2023

1.3K
Optimized LC-MS/MS Method for the High-throughput Analysis of Clinical Samples of Ivacaftor, Its Major Metabolites, and Lumacaftor in Biological Fluids of Cystic Fibrosis Patients06:14

Optimized LC-MS/MS Method for the High-throughput Analysis of Clinical Samples of Ivacaftor, Its Major Metabolites, and Lumacaftor in Biological Fluids of Cystic Fibrosis Patients

Published on: October 15, 2017

8.5K

Related Concept Videos

FDA Approved Drugs: Changes to Approved Drugs01:26

FDA Approved Drugs: Changes to Approved Drugs

Post-approval, manufacturers may modify an approved new or generic drug product. Such modifications can encompass alterations in the Active Pharmaceutical Ingredient (API), manufacturing process, formulation, batch size, manufacturing site, and container closure system (FDA Guidance for Industry, April 2004). Often, a drug product may undergo multiple changes.These modifications require careful evaluation to determine their potential impact on the drug product's identity, strength, quality,...
12
Drug Administration and Therapy Phases: Overview01:26

Drug Administration and Therapy Phases: Overview

Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
847
Bioequivalence studies: Biowaivers01:13

Bioequivalence studies: Biowaivers

Body:In certain scenarios, in vitro dissolution tests can replace in vivo bioequivalence studies. This is particularly true when a drug product, though available in varying strengths, maintains proportional similarity in its active and inactive ingredients. In such cases, the need for in vivo bioequivalence studies for lower strength variants may be waived, provided dissolution tests and in vivo studies on the highest strength yield satisfactory results.Bioequivalence can be indicated through...
15
Prescription, Nonprescription and Orphan Drugs01:02

Prescription, Nonprescription and Orphan Drugs

Prescription drugs require a prescription from a medical practitioner and can only be obtained from a pharmacy. They have many applications, including treating pain, anxiety, and hypertension.
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
897
Clinical Trials: Overview01:11

Clinical Trials: Overview

Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
3.7K
Drugs for Treatment of Diarrhea-Predominant IBS01:17

Drugs for Treatment of Diarrhea-Predominant IBS

Diarrhea-predominant irritable bowel syndrome (IBS-D) is a subtype of IBS characterized primarily by frequent, loose, or watery stools, abdominal pain, and abdominal discomfort. Therapeutic approaches to managing IBS-D include dietary changes, stress management techniques, and pharmaceutical interventions.
Two specific drugs used in the treatment are alosetron (Lotronex) and eluxadoline (Viberzi). Alosetron, a 5-HT3 antagonist, works by slowing the movement of stools in the gut, reducing bowel...
353