Betaine Anhydrous

Generic Name: betaine anhydrous

Methylating Agent [EPC]Over-the-Counter (OTC)

Brand Names:

Betaine Anhydrous

11. DESCRIPTION Betaine Anhydrous for Oral Solution is an agent for the treatment of homocystinuria. It contains no ingredients other than anhydrous betaine. Betaine Anhydrous for Oral Solution is a white, granular, hygroscopic powder, which is diluted in water and administered orally. The chemical name of betaine anhydrous powder is trimethylglycine. It has a molecular weight of 117.15. The structural formula is: Structural Formula

Overview

11. DESCRIPTION Betaine Anhydrous for Oral Solution is an agent for the treatment of homocystinuria. It contains no ingredients other than anhydrous betaine. Betaine Anhydrous for Oral Solution is a white, granular, hygroscopic powder, which is diluted in water and administered orally. The chemical name of betaine anhydrous powder is trimethylglycine. It has a molecular weight of 117.15. The structural formula is: Structural Formula

Uses

1. INDICATIONS AND USAGE Betaine Anhydrous for Oral Solution is indicated for the treatment of homocystinuria to decrease elevated homocysteine blood concentrations in pediatric and adult patients. Included within the category of homocystinuria are: • Cystathionine beta-synthase (CBS) deficiency • 5,10-methylenetetrahydrofolate reductase (MTHFR) deficiency • Cobalamin cofactor metabolism (cbl) defect Betaine Anhydrous for Oral Solution is a methylating agent indicated in pediatric and adult patients for the treatment of homocystinuria to decrease elevated homocysteine blood concentrations.

Dosage

2. DOSAGE AND ADMINISTRATION Adults and Pediatric Patients 3 Years of Age and Older • The recommended dosage is 6 grams per day, administered orally in divided doses of 3 grams twice daily. (2.1) Pediatric Patients Less than 3 Years of Age • The recommended starting dosage is 100 mg/kg/day, administered orally in divided doses of 50 mg/kg twice daily, and then increased weekly by 50 mg/kg increments. (2.1) • Monitor patient response by plasma homocysteine concentrations. (2.1) • Increase the dosage gradually until the plasma total homocysteine concentration is undetectable or present only in small amounts.

Side Effects

6. ADVERSE REACTIONS The following serious adverse reactions are described elsewhere in labeling: • Hypermethioninemia and cerebral edema in patients with CBS deficiency [see Warnings and Precautions (5.1)] . Most common adverse reactions (> 2%) are: nausea and gastrointestinal distress, based on physician survey (6.1). To report SUSPECTED ADVERSE REACTIONS, contact Eton Pharmaceuticals, Inc. at 1-855-224-0233 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . See 17 for PATIENT COUNSELING INFORMATION. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

Warnings

5. WARNINGS AND PRECAUTIONS 5.1 Hypermethioninemia in Patients with CBS Deficiency Patients with homocystinuria due to cystathionine beta-synthase (CBS) deficiency may also have elevated plasma methionine concentrations. Treatment with Betaine Anhydrous for Oral Solution may further increase methionine concentrations due to the remethylation of homocysteine to methionine. Cerebral edema has been reported in patients with hypermethioninemia, including patients treated with Betaine Anhydrous for Oral Solution [see Adverse Reactions (6.2)] . Monitor plasma methionine concentrations in patients with CBS deficiency. Plasma methionine concentrations should be kept below 1,000 micromol/L through dietary modification and, if necessary, a reduction of Betaine Anhydrous for Oral Solution dosage. 4. CONTRAINDICATIONS None. None

Storage

16. HOW SUPPLIED/STORAGE AND HANDLING Betaine Anhydrous for Oral Solution is available in plastic bottles containing 180 grams of betaine anhydrous as a white, granular, hygroscopic powder. Each bottle is equipped with a plastic child-resistant cap and is supplied with a polypropylene measuring scoop. One level scoop (1.5 cc) is equal to 1 gram of betaine anhydrous powder.

Frequently Asked Questions

What is Betaine Anhydrous used for?

1. INDICATIONS AND USAGE Betaine Anhydrous for Oral Solution is indicated for the treatment of homocystinuria to decrease elevated homocysteine blood concentrations in pediatric and adult patients. Included within the category of homocystinuria are: • Cystathionine beta-synthase (CBS) deficiency • 5,10-methylenetetrahydrofolate reductase (MTHFR) deficiency • Cobalamin cofactor metabolism (cbl) defect Betaine Anhydrous for Oral Solution is a methylating agent indicated in pediatric and adult patients for the treatment of homocystinuria to decrease elevated homocysteine blood concentrations.

What are the side effects of Betaine Anhydrous?

6. ADVERSE REACTIONS The following serious adverse reactions are described elsewhere in labeling: • Hypermethioninemia and cerebral edema in patients with CBS deficiency [see Warnings and Precautions (5.1)] . Most common adverse reactions (> 2%) are: nausea and gastrointestinal distress, based on physician survey (6.1). To report SUSPECTED ADVERSE REACTIONS, contact Eton Pharmaceuticals, Inc. at 1-855-224-0233 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . See 17 for PATIENT COUNSELING INFORMATION. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

What are the important warnings for Betaine Anhydrous?

5. WARNINGS AND PRECAUTIONS 5.1 Hypermethioninemia in Patients with CBS Deficiency Patients with homocystinuria due to cystathionine beta-synthase (CBS) deficiency may also have elevated plasma methionine concentrations. Treatment with Betaine Anhydrous for Oral Solution may further increase methionine concentrations due to the remethylation of homocysteine to methionine. Cerebral edema has been reported in patients with hypermethioninemia, including patients treated with Betaine Anhydrous for Oral Solution [see Adverse Reactions (6.2)] . Monitor plasma methionine concentrations in patients with CBS deficiency. Plasma methionine concentrations should be kept below 1,000 micromol/L through dietary modification and, if necessary, a reduction of Betaine Anhydrous for Oral Solution dosage. 4. CONTRAINDICATIONS None. None

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Medical Disclaimer

This drug information is for educational purposes only and should not replace professional medical advice. Drug information is sourced from the FDA National Drug Code Directory and Structured Product Labeling. Always consult with a healthcare provider before starting, stopping, or changing any medication.