Miglustat

Niemann-Pick Disease Type C

ActelionSmall moleculePhase 4 / ApprovedDormant

BBB

76/ 100

Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence: 63/ 100
Competitive Position: 95/ 100

Competitive context

3 Active assets in the Niemann-Pick Disease Type C cohort.

Asset / Sponsor	Phase	Rating
az-3102 Azafaros B.V.	Phase 3	BB
Nizubaglustat Azafaros B.V.	Phase 3	B
N-Acetyl-L-Leucine IntraBio Inc	Phase 3	B
2-HYDROXYPROPYL BETA-CYCLODEXTRIN (DS 4.2) Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company	Phase 2/3	B
Arimoclomol ZevraDenmark	Phase 2/3	CCC

View all 11 →

Same molecule, other indications

Miglustat is also rated in:

Gaucher disease type IDormant

BB

Indicative ·

64 / 100

cystic fibrosisDormant

B

Indicative ·

63 / 100

glycogen storage disease due to acid maltase deficiency, late-onsetDormant

B

Indicative ·

62 / 100

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