CYSTAGON (cysteamine bitartrate)
Cystagon helps children and adults who have nephropathic cystinosis, a metabolic condition where cystine builds up and damages organs like the kidneys. This medication is used to manage the accumulation of these crystals, which otherwise lead to serious issues like renal failure, growth problems, and vision sensitivity. It is a critical therapy for preventing the progressive damage to the muscles, eyes, and central nervous system associated with the disease.
How CYSTAGON Works
This drug works by entering the lysosomes within cells to trigger a chemical reaction with stored cystine. It converts the trapped cystine into two different substances—cysteine and a cysteine-cysteamine disulfide—that are able to exit the lysosome. This process prevents the toxic accumulation of cystine crystals that would otherwise damage the kidneys and other vital organs.
Development Insights
Details
- Status
- Prescription
- First Approved
- 1994-08-15
- Routes
- ORAL
- Dosage Forms
- CAPSULE
CYSTAGON Approval History
What CYSTAGON Treats
1 indicationsCYSTAGON is approved for 1 conditions since its original approval in 1994. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Nephropathic Cystinosis
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Clinical Trial Registry
4 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT05994534 INCA | C-23-01 | Ph 1, Ph 2 | recruiting | PK and PD Study of NPI-001 and Cysteamine Bitartrate |
| NCT02023866 MITO-001 results posted | RP103-MITO-001 | Ph 2 | completed | Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease |
| NCT02473445 results posted | RP103-MITO-002 | Ph 2 | terminated | A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease |
| NCT03883984 | Hershey-101 | Ph 1 | completed | Cysteamine for Asthma |
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.
CYSTAGON FDA Label Details
Indications & Usage
FDA Label (PDF)CYSTAGON is indicated for the treatment of Nephropathic Cystinosis.
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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