How many drugs treat Cholestanol storage disease?

Drug Landscape tracks 2 marketed treatments for Cholestanol storage disease (deduplicated by molecule). 0 Phase 3 candidates and 0 Phase 2 candidates are in the pipeline.

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Subscribe to /feed/disease/cholestanol-storage-disease.xml in Feedly / Inoreader. Every regulatory action, PDUFA date, or trial completion in this treatment area lands in your feed automatically.

Last reviewed 2026-06-10 · How we verify

Cholestanol storage disease — Treatment Landscape & Competitive Intelligence

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Cholestanol storage disease (disease) competitive landscape: 2 marketed treatments tracked, 0 Phase 3 candidates, 0 Phase 2 candidates. Recent regulatory actions and upcoming PDUFA dates across the entire treatment set.

2 marketed 0 Phase 3 0 Phase 2 Live · 30-min refresh

Marketed treatment landscape

Approved drugs treating Cholestanol storage disease, deduplicated by molecule. See the full disease page for line-of-therapy detail.

Drug	Generic	Sponsor	Class	Target	Line of therapy	First approval
Cholbam	CHOLIC ACID	Mirum	Bile Acid [EPC]	G-protein coupled bile acid receptor 1		2015-01-01
Cholbam	Cholbam	Mirum Pharmaceuticals, Inc.		G-protein coupled bile acid receptor 1, Bile acid receptor

Phase 3 pipeline

No Phase 3 pipeline candidates tracked.

Phase 2 pipeline

No Phase 2 pipeline candidates tracked.

Recent regulatory actions (last 90 days)

No regulatory actions in the last 90 days for this treatment set.

Upcoming PDUFA dates (next 180 days)

No PDUFA dates in the next 180 days for this treatment set.

Sponsor landscape

Mirum · 1 drug in Cholestanol storage disease
Mirum Pharmaceuticals, Inc. · 1 drug in Cholestanol storage disease

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Every new regulatory action, PDUFA date, or trial completion in Cholestanol storage disease:

Cite this brief

Drug Landscape (2026). Cholestanol storage disease — Treatment Landscape & Competitive Intelligence Brief. https://druglandscape.com/ci/disease/cholestanol-storage-disease. Accessed 2026-06-10.

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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing