Betaine

Betaine is an FDA-registered drug product whose active ingredient is BETAINE. It is labelled by Cosette Pharmaceuticals, Inc. taken by oral and marketed as a human prescription drug product. Identifiers on file: NDC 0713-0352, application ANDA214864, label effective 20260520. It is one of 3 FDA drug label records sharing the active ingredient BETAINE. This page reproduces the FDA-approved label text so you can check what a product is, who makes it and how it is supplied before discussing it with a pharmacist or prescriber.

Across FDA labelling, products containing BETAINE are most often described in connection with: betaine anhydrous, oral solution, treatment homocystinuria, decrease elevated, homocysteine blood, adult patients, included within, cystathionine beta-synthase. Wording differs between labels, and the full indications section on this page takes precedence when it is present.

1 INDICATIONS & 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. Included within the category of homocystinuria are: Cystathionine beta-synthase (CBS) deficiency 5,10-methylenetetrahydrofolate reductase (MTHFR) deficiency Cobalamin cofactor metabolism (cbl) defect

2 DOSAGE & 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. (2.1) Preparation and Administration Instructions Prescribed amount of Betaine Anhydrous for Oral Solution should be measured with the measuring scoop provided and then dissolved in 4 to 6 ounces of water, juice, milk, or formula until completely dissolved, or mixed with food for immediate ingestion. (2.2) 2.1 Dosage Therapy with Betaine Anhydrous for Oral Solution should be directed by physicians knowledgeable in the management of patients with homocystinuria. 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. Pediatric Patients Less than 3 Years of Age The recommended starting dosage is 100 mg/kg/day divided in twice daily doses, and then increased weekly by 50 mg/kg increments. Monitoring Monitor patient response to Betaine Anhydrous for Oral Solution by homocysteine plasma concentration. Increase the dosage in all patients gradually until the plasma total homocysteine concentration is undetectable or present only in small amounts. An initial response in homocysteine plasma concentrations usually occurs within several days and steady state plasma concentrations occur within a month. Monitor plasma methionine concentrations in patients with CBS deficiency [see Warnings and Precautions (5.1)]. Maximum Dosage Dosages of up to 20 grams/day have been necessary to control homocysteine concentrations in some patients. However, one pharmacokinetic and pharmacodynamic in vitro simulation study indicated minimal benefit from exceeding a twice-daily dosing schedule and a 150 mg/kg/day dosage for Betaine Anhydrous for Oral Solution. 2.2 Preparation and Administration Instructions Shake bottle lightly before removing cap. Measure the number of scoops for the patient's dose with the scoop provided. One level scoop (1.9cc) is equivalent to 1 gram of betaine anhydrous powder. Mix powder with 4 to 6 ounces (120 to 180 mL) of water, juice, milk, or formula until completely dissolved, or mix with food, then ingest mixture immediately. Always replace the cap tightly after using and protect the bottle from moisture.

4 CONTRAINDICATIONS None. None (4)

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 Cosette Pharmaceuticals, Inc. at 1-800-922-1038 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 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. The assessment of clinical adverse reactions is based on a survey study of 41 physicians, who treated a total of 111 homocystinuria patients with Betaine Anhydrous for Oral Solution. Adverse reactions were retrospectively recalled and were not collected systematically in this open-label, uncontrolled, physician survey. Thus, this list may not encompass all types of potential adverse reactions, reliably estimate their frequency, or establish a causal relationship to drug exposure. The following adverse reactions were reported (Table 1): Table 1: Number of Patients with Adverse Reactions to Betaine Anhydrous for Oral Solution by Physician Survey Adverse Reactions Number of Patients Nausea 2 Gastrointestinal distress 2 Diarrhea 1 "Bad Taste" 1 "Caused Odor" 1 Questionable psychological changes 1 "Aspirated the powder" 1 6.2 Post-marketing Experience The following adverse reactions have been identified during post approval use of Betaine Anhydrous for Oral Solution. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure. Severe cerebral edema and hypermethioninemia have been reported within 2 weeks to 6 months of starting Betaine Anhydrous for Oral Solution therapy, with complete recovery after discontinuation of Betaine Anhydrous for Oral Solution. All patients who developed cerebral edema had homocystinuria due to CBS deficiency and had severe elevation in plasma methionine concentrations (range 1,000 to 3,000 microM). As cerebral edema has also been reported in patients with hypermethioninemia, secondary hypermethioninemia due to betaine therapy has been postulated as a possible mechanism of action [ see Warnings and Precautions (5.1)] . Other adverse reactions include: anorexia, agitation, depression, irritability, personality disorder, sleep disturbed, dental disorders, diarrhea, glossitis, nausea, stomach discomfort, vomiting, hair loss, hives, skin odor abnormalities, and urinary incontinence.

FDA lists 3 drug label records with BETAINE as the active ingredient. The most common dosage forms and the manufacturers with the most records are summarised below; strengths, pack sizes and inactive ingredients can still differ between apparently similar products.

1 FDA recall record(s) match this product name or manufacturer. A match means the firm or product name appears in the enforcement report; confirm lot numbers and dates against the official FDA notice before acting.

What is Betaine used for?
1 INDICATIONS & 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 …

Who makes Betaine?
Cosette Pharmaceuticals, Inc. is the labeler of record for Betaine. 3 FDA label records share the active ingredient BETAINE, so generic and branded versions may come from different companies; the manufacturer named on your pack is the one that matters for any enquiry.

What form does Betaine come in?
administered by oral. Strengths and pack sizes vary by label, so compare the NDC on your pack with the identifier listed on this page.

Is Betaine prescription or over-the-counter?
The FDA label for this product is recorded as human prescription drug. Marketing status can differ between strengths and countries, so check the dispensing rules where you live.

Are there other products containing BETAINE?
Yes. 3 FDA drug label records list BETAINE as the active ingredient. Products with the same active ingredient can usually be substituted only when a prescriber or pharmacist confirms it is appropriate.

Has Betaine been recalled?
1 FDA recall record(s) match this product name or manufacturer. Review the recall and safety notices section on this page and confirm lot numbers against the official FDA enforcement report.

Where does this information come from?
All content is reproduced from FDA Structured Product Labelling data via openFDA. It is provided for identification and reference only and is not medical advice.