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 13h ago · AI intelligence enriched 4d ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
OPFOLDA (miglustat) is an oral glucosylceramide synthase inhibitor approved in September 2023 for lysosomal storage disorders. It works by reducing the synthesis of glucosylceramide, a substrate that accumulates in these rare genetic diseases. The drug represents a substrate reduction therapy approach for patients with gaucher disease and other sphingolipidoses.
Growth12.0 years to LOE
Early-stage growth product with zero current competitive pressure, indicating opportunity for market development teams to establish market presence and build formulary relationships.
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 offers entry into the rare disease specialty pharmaceuticals sector with Amicus Therapeutics, a focused rare disease company. The early-stage growth lifecycle and zero-competitor landscape creates opportunity to build market-from-scratch programs and establish thought leadership in lysosomal storage disorders.
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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.