NDAORALCAPSULE
Approved
Jul 2003
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
3
Data completeness
5/8 — Partial
ZAVESCA (miglustat) by Johnson & Johnson is glucosylceramide synthase inhibitors [moa]. Approved for pompe disease. First approved in 2003.
Drug data last refreshed 18h ago · AI intelligence enriched 2w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ZAVESCA (miglustat) is an oral glucosylceramide synthase inhibitor approved in 2003 for treating lysosomal storage disorders, specifically Pompe Disease and Batten Disease. It works by inhibiting the production of glucosylceramide, reducing the accumulation of storage material in cells. This small-molecule approach addresses the underlying metabolic dysfunction in these rare genetic conditions.
$0.002BPart D
LOE ApproachingDeclining
Modest Part D footprint with 67 annual claims signals a niche rare disease product entering LOE phase, likely requiring defensive positioning and potential transition planning for commercial teams.
Mechanism of Action
Glucosylceramide Synthase Inhibitors
Pharmacologic Class:
Glucosylceramide Synthase Inhibitor
Indications (1)
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
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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.