Miglustat

Cystic Fibrosis

Assistance Publique - Hôpitaux de ParisSmall moleculePhase 2/3Dormant

B

63/ 100

Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence: 58/ 100
Competitive Position: 73/ 100

Competitive context

82 Active assets in the Cystic Fibrosis cohort.

Asset / Sponsor	Phase	Rating
Tezacaftor Vertex Pharmaceuticals Incorporated	Phase 4	A
Ivacaftor Meyer Children's Hospital IRCCS	Phase 4	BBB
Lumacaftor Hospices Civils de Lyon	Phase 4	BBB
Liprotamase Anthera Pharmaceuticals	Phase 4	BBB
ELEXACAFTOR Meyer Children's Hospital IRCCS	Phase 4	BBB

View all 50 →

Same molecule, other indications

Miglustat is also rated in:

Niemann-Pick disease type CDormant

BBB

Indicative ·

76 / 100

Gaucher disease type IDormant

BB

Indicative ·

64 / 100

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

B

Indicative ·

62 / 100

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