CYSTADANE Powder for oral solution Ref.[10796] Active ingredients: Betaine

2.1 Dosage

Therapy with CYSTADANE 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 CYSTADANE by homocysteine plasma concnetration. 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 CYSTADANE.

2.2 Prepartion 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 (1.7 mL) 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.

There is no information on CYSTADANE overdose in humans. In an acute toxicology study in rats, death occurred frequently at doses equal to or greater than 10 g/kg.

Store at room temperature, 15 to 30˚C (59 to 86˚F). Protect from moisture.