NDAORALCAPSULE
Approved
Sep 2023
Lifecycle
Growth
Competitive Pressure
0/100
Clinical Trials
2
Data completeness
4/8 — Partial
OPFOLDA (miglustat) by Amicus Therapeutics is glucosylceramide synthase inhibitors [moa]. First approved in 2023.
Drug data last refreshed 5h 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 rare lysosomal storage disorders. It works by reducing glucosylceramide accumulation in cells, addressing the underlying pathophysiology of these genetic diseases. The drug represents a substrate reduction therapy approach to managing life-limiting metabolic conditions.
Growth12.2 years to LOE
Early growth-stage product with significant market development runway; commercial teams are building market access and patient identification infrastructure.
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 (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
Career Relevance
Working on OPFOLDA positions you in the high-value rare disease segment with long-term commercial stability and strategic importance to Amicus Therapeutics. The growth-stage lifecycle and low competitive intensity create opportunities for visibility, account ownership, and influence on product strategy during the critical market-building phase.
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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.