Betaine anhydrous
Orphan DrugFDA Approved
Description
Betaine anhydrous is an orphan drug used primarily for homocystinuria treatment. While not directly indicated for ataxia with oculomotor apraxia type 1, it has been investigated for neurological conditions involving metabolic dysfunction and oxidative stress. The compound acts as a methyl donor in homocysteine metabolism and has neuroprotective properties.
Indications & Therapeutic Use
Homocystinuria, hyperhomocysteinemia, metabolic disorders (investigational for neurological conditions)
Linked Diseases:
Global Availability (8 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Betaine anhydrous
| Generic Name | Betaine anhydrous |
| Brands | 1 brand available |
| Active Ingredient | Betaine anhydrous |
| Drug Class | Homocystinuria |
| Manufacturer | Recordati Rare Diseases |
| Dosage Forms | Oral powder for solution 1g/1.7mL |
| Medical Code | A16AA06 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 21 days |
| Reg. Status | FDA Approved |
| Countries | 8 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations9 Validated Nodes