Source: Medicines & Healthcare Products Regulatory Agency (GB) Revision Year: 2017 Publisher: Orphan Europe SARL, Immeuble Le Wilson, 70, Avenue du General de Gaulle, F-92 800 Puteaux, France
Adjunctive treatment of homocystinuria, involving deficiencies or defects in:
Cystadane should be used as supplement to other therapies such as vitamin B6 (pyridoxine), vitamin B12 (cobalamin), folate and a specific diet.
Cystadane treatment should be supervised by a physician experienced in the treatment of patients with homocystinuria.
The recommended total daily dose is 100 mg/kg/day given in 2 doses daily. However, the dose should be individually titrated according to plasma levels of homocysteine and methionine. In some patients doses above 200 mg/kg/day were needed to reach therapeutic goals. Caution should be exercised with up-titrating doses for patients with CBS deficiency due to the risk for hypermethioninaemia. Methionine levels should be closely monitored in these patients.
Experience with betaine anhydrous therapy in patients with renal insufficiency or non-alcoholic hepatic steatosis has demonstrated no need to adapt the dose regimen of Cystadane.
The bottle should be lightly shaken before opening. Three measuring spoons are provided which dispense either 100 mg, 150 mg or 1 g of betaine anhydrous. It is recommended that a heaped measuring spoon is removed from the bottle and a flat surface e.g. base of a knife is drawn across the top of the measure. This will give the following doses: small measure 100 mg, middle size measure 150 mg and large measure 1 g of betaine anhydrous.
The powder should be mixed with water, juice, milk, formula or food until completely dissolved and ingested immediately after mixing.
The aim of treatment is to keep plasma levels of total homocysteine below 15 µM or as low as possible. The steadystate response usually occurs within a month.
No case of overdose has been reported.
Unopened bottle: 3 years.
After the first opening: 3 months.
Do not store above 25°C.
Keep the bottle tightly closed in order to protect from moisture.
For storage conditions after first opening of the medicinal product, see section 6.3.
HDPE bottles with a child resistant closure.
Each pack contains 1 bottle with 180 g of powder and three measuring spoons.
Any unused medicinal product or waste material should be disposed of in accordance with local requirements.
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