Overview
11 DESCRIPTION CYSTADANE (betaine anhydrous for oral solution) is an agent for the treatment of homocystinuria. It contains no ingredients other than anhydrous betaine. CYSTADANE 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.
Mechanism of Action
12 CLINICAL PHARMACOLOGY 12.1 Mechanism of Action CYSTADANE acts as a methyl group donor in the remethylation of homocysteine to methionine in patients with homocystinuria. Betaine occurs naturally in the body. It is a metabolite of choline and is present in small amounts in foods such as beets, spinach, cereals, and seafood. 12.2 Pharmacodynamics CYSTADANE was observed to lower plasma homocysteine concentrations in three types of homocystinuria, including CBS deficiency; MTHFR deficiency; and cbl defect. Patients have taken CYSTADANE for many years without evidence of tolerance. There has been no demonstrated correlation between Betaine concentrations and homocysteine concentrations. In CBS-deficient patients, large increases in methionine concentrations over baseline have been observed.
Indications
- 1 INDICATIONS AND USAGE CYSTADANE ® 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 CYSTADANE is a methylating agent indicated in pediatric and adult patients for the treatment of homocystinuria to decrease elevated homocysteine blood concentrations.
Common Doses
- 100 mg
- 50 mg
- 150 mg
- 1.7 mL
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.
Contraindications
- 4 CONTRAINDICATIONS None. None ( 4 )
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 Recordati Rare Diseases Inc. at 1-888-575-8344, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.
Interactions
- No major interactions listed on the FDA label. Always consult a healthcare professional.