MIGLUSTAT
Miglustat treats adults with mild to moderate type 1 Gaucher disease who cannot receive standard enzyme replacement therapy. It is used as a monotherapy for patients who may have allergies, hypersensitivities, or poor venous access that prevents them from using other treatments. This medication provides an alternative option for managing the disease when traditional enzyme-based approaches are not feasible.
How MIGLUSTAT Works
Miglustat works by inhibiting glucosylceramide synthase, the enzyme responsible for the initial step in glycosphingolipid production. By slowing down the synthesis of these lipids, the drug reduces the amount of substrate that builds up in the body. This allows the patient's remaining natural enzymes to more effectively break down the existing glucosylceramide.
Details
- Status
- Discontinued
- First Approved
- 2018-04-17
- Routes
- ORAL
- Dosage Forms
- CAPSULE
MIGLUSTAT Approval History
What MIGLUSTAT Treats
1 indicationsMIGLUSTAT is approved for 1 conditions since its original approval in 2018. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Gaucher Disease
MIGLUSTAT Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to MIGLUSTAT
3 of 4FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
9 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT04808505 ROSSELLA | ATB200-08 | Ph 3 | recruiting | A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 |
| NCT03911505 | ATB200-04 | Ph 3 | active not recruiting | ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD |
| NCT03822013 EMTISTD | 97-01-30-36953 | Ph 3 | terminated | Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) |
| NCT03729362 PROPEL results posted | ATB200-03 | Ph 3 | completed | A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease |
| NCT03910621 | AC-056C405 | Ph 4 | completed | Safety and Efficacy of Miglustat in Chinese NPC Patients |
| NCT02030015 Syner-G results posted | Syner_G_Regimen U54NS065768, 1311M46101 | Ph 4 | terminated | Synergistic Enteral Regimen for Treatment of the Gangliosidoses |
| NCT00742092 | AC-056A202 | Ph 2 | completed | Miglustat in Cystic Fibrosis |
| NCT01760564 | 200802043M | Ph 3 | completed | Application of Miglustat in Patients With Niemann-Pick Type C |
| NCT00945347 | MIG-99 | Ph 2 | completed | Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ? |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
MIGLUSTAT FDA Label Details
Indications & Usage
MIGLUSTAT is indicated for the treatment of Gaucher Disease.
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Full clinical data, patents, trials, and competitive landscape for miglustat.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.