YARGESA (miglustat)
Yargesa treats adults with mild to moderate type 1 Gaucher disease. It is used for patients who cannot receive standard enzyme replacement therapy due to clinical challenges such as allergies, hypersensitivity, or poor venous access. This medication provides a monotherapy option for managing the condition when other primary treatments are not a viable choice.
How YARGESA Works
Yargesa works by inhibiting glucosylceramide synthase, the enzyme responsible for the first step in the synthesis of most glycosphingolipids. By slowing the production of these lipids, the drug reduces the amount of substrate that the body's deficient enzymes must break down. This substrate reduction therapy allows the patient's residual enzyme activity to function more effectively.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2020-08-06
- Routes
- ORAL
- Dosage Forms
- CAPSULE
YARGESA Approval History
What YARGESA Treats
1 indicationsYARGESA is approved for 1 conditions since its original approval in 2020. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Gaucher Disease
YARGESA 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 YARGESA
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.
YARGESA FDA Label Details
Indications & Usage
YARGESA is indicated for the treatment of Gaucher Disease.
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment