NDAORALCAPSULE
Approved
Jul 2003
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
3
Data completeness
4/8 — Partial
ZAVESCA (miglustat) by Johnson & Johnson is glucosylceramide synthase inhibitors [moa]. First approved in 2003.
Drug data last refreshed 6h ago · AI intelligence enriched Yesterday
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ZAVESCA (miglustat) is an oral glucosylceramide synthase inhibitor approved in 2003 for rare lysosomal storage disorders, specifically Gaucher disease type 1 and Niemann-Pick disease type C. It works by reducing the synthesis of glucosylceramide, a lipid that accumulates in cells of affected patients. The drug represents a foundational small-molecule approach to enzyme replacement therapy in orphan indications.
$0.002BPart D
LOE ApproachingMinimal commercial footprint with ultra-rare indication positioning; career growth limited to specialized orphan disease or medical affairs tracks.
Mechanism of Action
Glucosylceramide Synthase Inhibitors
Pharmacologic Class:
Glucosylceramide Synthase Inhibitor
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (3)
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Started Jul 2023
36 enrolled
Glycogen Storage Disease Type II Infantile Onset
An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease
Started Mar 2022
6 enrolled
Batten Disease
A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction
Started Oct 2016
2 enrolled
Pompe DiseaseHypersensitivity Reaction
Career Relevance
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.