BETAINE
Betaine is a medication used for both children and adults who have homocystinuria. It helps patients with this condition by lowering high levels of homocysteine in the blood. This includes people with specific genetic issues like CBS deficiency, MTHFR deficiency, or certain cobalamin metabolism defects.
How BETAINE Works
This drug works by acting as a methyl group donor. It assists in the process of turning homocysteine back into methionine, which helps manage the chemical balance in patients with homocystinuria. Betaine is a naturally occurring substance in the body and can also be found in foods like beets and spinach.
Details
- Status
- Prescription
- First Approved
- 2021-11-23
- Routes
- ORAL
- Dosage Forms
- FOR SOLUTION
BETAINE Approval History
What BETAINE Treats
4 indicationsBETAINE is approved for 4 conditions since its original approval in 2021. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Homocystinuria
- Cystathionine Beta-Synthase Deficiency
- Methylenetetrahydrofolate Reductase Deficiency
- Cobalamin Cofactor Metabolism Defect
BETAINE 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 BETAINE
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.
BETAINE FDA Label Details
Indications & Usage
BETAINE is indicated for the treatment of Homocystinuria; Cystathionine Beta-Synthase Deficiency; Methylenetetrahydrofolate Reductase Deficiency; Cobalamin Cofactor Metabolism Defect.
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Full clinical data, patents, trials, and competitive landscape for betaine.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.