CYSTADANE (betaine)
CYSTADANE is indicated for the treatment of Homocystinuria; Cystathionine Beta-Synthase Deficiency; Methylenetetrahydrofolate Reductase Deficiency; Cobalamin Cofactor Metabolism Defect.
Development Insights
Details
- Status
- Prescription
- First Approved
- 1996-10-25
- Routes
- ORAL
- Dosage Forms
- FOR SOLUTION
CYSTADANE Approval History
What CYSTADANE Treats
4 indicationsCYSTADANE is approved for 4 conditions since its original approval in 1996. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Homocystinuria
- Cystathionine Beta-Synthase Deficiency
- Methylenetetrahydrofolate Reductase Deficiency
- Cobalamin Cofactor Metabolism Defect
CYSTADANE Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
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 CYSTADANE
FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
6 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT07276204 | Betaine for MASH | Ph 2 | not yet recruiting | Betaine vs. Placebo for Serologically Diagnosed Metabolic Dysfunction-associated Steatohepatitis (MASH) |
| NCT06308367 | XWTEBS | Ph 2 | recruiting | The Therapeutic Effect of Betaine in Syringomyelia |
| NCT02404337 HCTBETAINE | P130908 | Ph 2 | completed | Betaine METABOLISM OF PATIENTS With Homocystinuria |
| NCT01950039 results posted | 2013P001265 7-13-CE-17 | Ph 2 | completed | Metabolic Effects of Betaine Supplementation |
| NCT01838941 results posted | RPGDN001 | Ph 3 | completed | Betaine and Peroxisome Biogenesis Disorders |
| NCT01749982 CABS results posted | AAAJ1959 | Ph 4 | completed | A Preliminary Study of Choline and Betaine Supplementation Among Adults Exposed to Arsenic in Bangladesh |
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.
CYSTADANE FDA Label Details
Indications & Usage
FDA Label (PDF)CYSTADANE is indicated for the treatment of Homocystinuria; Cystathionine Beta-Synthase Deficiency; Methylenetetrahydrofolate Reductase Deficiency; Cobalamin Cofactor Metabolism Defect.
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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