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)

Global Availability (8 countries)

CountryAccess RouteRegulatory PathwayLead TimeStatus

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Available Through

This drug is available for procurement through the following fulfilment partner.

Fulfilment Partner
Betaine anhydrous
Generic NameBetaine anhydrous
Brands1 brand available
Active IngredientBetaine anhydrous
Drug ClassHomocystinuria
ManufacturerRecordati Rare Diseases
Dosage FormsOral powder for solution 1g/1.7mL
Medical CodeA16AA06
Orphan StatusYes — Orphan Drug
Cold ChainNot Required
Lead Time21 days
Reg. StatusFDA Approved
Countries8 countries
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations9 Validated Nodes