Cholbam

(Cholic acid)

Phase 2Small molecule

MMirum Pharmaceuticals, Inc.

Cholbam is one of 18 small molecule programs targeting Hepatic steatosis. It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[3][1]

Indication

Hepatic SteatosisActive, not recruiting

Smith-Lemli-Opitz SyndromeActive, not recruiting

Hepatic SteatosisPhase 2

Active, not recruiting

Smith-Lemli-Opitz SyndromePhase 1/Phase 2

Active, not recruiting

Competition

Competitive Landscape

18 competitors in Hepatic Steatosis

#DrugCompanyPhaseTrials

Actos

Epidiolex

Farxiga

Pletal

Trental

VValidus Pharmaceuticals

Selzentry

1 7

Metadoxine

AAlcobra Ltd.

Approved

1 8

Rimonabant

Glucophage

1 10

Aquasol E

NNational Eye Institute (NEI)

Phase 2

—

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Clinical

Trials by Indication[1]

Children1 trial

Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis

Completed·MMirum Pharmaceuticals, Inc.·Phase 3·NCT01115582·16 enrolled

Peroxisomal Disorders1 trial

Study of Bile Acids in Patients With Peroxisomal Disorders

Terminated·UUniversity of Cincinnati·N/A·NCT00004442·25 enrolled

Hepatic steatosis1 trial

Cholic Acid for Hepatic Steatosis in Lipodystrophy

Completed·UUniversity of Texas Southwestern Medical Center·Phase 2·NCT00457639·18 enrolled

Metabolism1 trial

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Completed·MMirum Pharmaceuticals, Inc.·Phase 3·NCT00007020·85 enrolled

Smith-Lemli-Opitz syndrome1 trial

Smith-Lemli-Opitz Syndrome and Cholic Acid

Completed·UUniversity of Nebraska·Phase 1/2·NCT03720990·12 enrolled

1 more indication

Unspecified1 trial

Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis

Completed·MMirum Pharmaceuticals, Inc.·Phase 3·NCT01438411·53 enrolled

Regulatory

FDA Approval Timeline[4]

Mar 17, 2015

CHOLBAM

NDA NDA205750

Sources

References

[1]
ClinicalTrials.gov — 6 trial records for Cholic acid. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Cholic acid compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Cholic acid. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA205750). U.S. Food & Drug Administration.
[5]
SEC EDGAR — Mirum Pharmaceuticals, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: MMirum Pharmaceuticals, Inc.

Cholbam

Status by Indication[3][1]

Competitive Landscape

Trials by Indication[1]

Children1 trial

Peroxisomal Disorders1 trial

Hepatic steatosis1 trial

Metabolism1 trial

Smith-Lemli-Opitz syndrome1 trial

Unspecified1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

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