How many drugs treat Niemann-Pick disease, type C?

Drug Landscape tracks 1 marketed treatments for Niemann-Pick disease, type C (deduplicated by molecule). 0 Phase 3 candidates and 0 Phase 2 candidates are in the pipeline.

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Last reviewed 2026-06-10 · How we verify

Niemann-Pick disease, type C — Treatment Landscape & Competitive Intelligence

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

Niemann-Pick disease, type C (disease) competitive landscape: 1 marketed treatments tracked, 0 Phase 3 candidates, 0 Phase 2 candidates. Recent regulatory actions and upcoming PDUFA dates across the entire treatment set.

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

Marketed treatment landscape

Approved drugs treating Niemann-Pick disease, type C, deduplicated by molecule. See the full disease page for line-of-therapy detail.

Drug	Generic	Sponsor	Class	Target	Line of therapy	First approval
Zavesca	MIGLUSTAT	AstraZeneca	Glucosylceramide Synthase Inhibitor [EPC]	Lysosomal alpha-glucosidase		2003-01-01

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

AstraZeneca · 1 drug in Niemann-Pick disease, type C

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Every new regulatory action, PDUFA date, or trial completion in Niemann-Pick disease, type C:

Cite this brief

Drug Landscape (2026). Niemann-Pick disease, type C — Treatment Landscape & Competitive Intelligence Brief. https://druglandscape.com/ci/disease/niemann-pick-disease-type-c. Accessed 2026-06-10.

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