Treats mild/moderate type 1 Gaucher disease. Sold as Zavesca, Opfolda. Every FDA application for this active ingredient: 2 branded and 4 generic.
As of Nov 15, 2026, FDA lists 6 applications for Miglustat (sold as Zavesca, Opfolda), first approved Jul 31, 2003, with 0 new uses approved and 0 published complete response letters.
| Miglustat | Part D | $5.3M | +16% | 26 |
| Zavesca | Part D | $1.3M | -7% | 0 |
| Yargesa | Part D | $638K | New | 0 |
| Opfolda | Part D | $59K | +45% | 25 |
| Study With Batten-1 (Miglustat Liquid Formulation) to Treat Ceroid Lipofuscinosis Neuronal 3 (CLN3) DiseaseCLN3 · Beyond Batten Disease Foundation | Phase 3 | Not yet recruiting | Oct 20, 2026 |
| A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C DiseaseGM2 Gangliosidosis, Niemann-Pick Type C Disease · Azafaros B.V. | Phase 2 | Active, not recruiting | Feb 10, 2026 |
| A Global Prospective Observational Registry of Patients With Pompe DiseasePompe Disease · Amicus Therapeutics | — | Recruiting | Nov 7, 2023 |
| A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18Glycogen Storage Disease Type II Infantile Onset · Amicus Therapeutics | Phase 3 | Recruiting | Mar 22, 2021 |
| ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDPompe Disease (Late-onset) · Amicus Therapeutics | Phase 3 | Active, not recruiting | Apr 11, 2019 |
| Jul 31, 2003 | ZavescaNDA 21348 · New molecular entity | Actelion | 7 | Marketed |
| Apr 17, 2018 | MiglustatANDA 208342 | Ani Pharms | 2 | Marketed |
| Aug 6, 2020 | YargesaANDA 209821 | Edenbridge Pharms | 1 | Marketed |
| Feb 3, 2022 | MiglustatANDA 209325 | Chartwell Rx | 0 | Discontinued |
| Sep 28, 2023 | OpfoldaNDA 215211 · New formulation or manufacturer | Amicus Therap US | 1 | Marketed |
| Mar 14, 2025 | MiglustatANDA 219111 | Navinta LLC | 0 | Marketed |
Applications, approvals and products come from FDA's Drugs@FDA data files, refreshed weekly, grouped by active ingredient as FDA spells it. Complete response letters are FDA's published letters. Medicare spending is CMS's Part D and Part B dashboard, matched to this ingredient by generic name; shortages are FDA's shortage list, matched by application number; clinical trials are ClinicalTrials.gov studies naming the ingredient as an intervention, refreshed daily; safety programs are FDA's REMS list, matched by application number or, for shared programs, by ingredient. Drugs@FDA
