NDAORALCAPSULE
Approved
Sep 2023
Lifecycle
Growth
Competitive Pressure
0/100
Clinical Trials
2
Data completeness
5/8 — Partial
OPFOLDA (miglustat) by Amicus Therapeutics is glucosylceramide synthase inhibitors [moa]. Approved for pompe disease. First approved in 2023.
Drug data last refreshed 21h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
OPFOLDA (miglustat) is an oral glucosylceramide synthase inhibitor approved in September 2023 for Pompe Disease, a rare inherited lysosomal storage disorder. It works by reducing the synthesis of glucosylceramide, thereby decreasing substrate accumulation in cells. This small-molecule approach offers an alternative mechanism to enzyme replacement therapies in the Pompe disease treatment landscape.
Growth12.4 years to LOE
As a newly approved (2023) growth-stage product in a rare disease market with minimal current competition, the brand team is likely lean but expanding with potential for headcount growth in commercial, medical affairs, and market access roles.
Mechanism of Action
Glucosylceramide Synthase Inhibitors
Pharmacologic Class:
Glucosylceramide Synthase Inhibitor
Indications (1)
Clinical Trials (2)
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
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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.