What is Cystadane?
Cystadane is an oral powder that contains the active substance betaine anhydrous.
What is Cystadane used for?
Cystadane is used in the treatment of homocystinuria, as a supplement to other therapies, such as vitamin B6 (pyridoxine), vitamin B12 (cobalamin), folate, and a specific diet.
Homocystinuria is a hereditary (genetic) disease in which the body is unable to completely metabolize the amino acid methionine. Methionine is naturally present in dietary proteins and is usually transformed into homocysteine, then into cysteine. Affected patients from homocystinuria they are unable to transform homocysteine into cysteine; consequently, homocysteine accumulates in the blood and urine. Typical symptoms of homocystinuria include: thrombosis (formation of blood clots in blood vessels), bone fragility, skeletal abnormalities, ectopia lentis (an abnormality in which the lens is displaced from its normal position) and mental retardation. This is a serious disease. with a high mortality rate. Cystadane is used in patients with all three known types of homocystinuria, caused by a deficiency in the substances necessary for the metabolism of methionine ("cystathionine beta-synthase" [CBS] or "5.10 - methylenetetrahydrofolate reductase "[MTHFR]) or from defects in the" metabolism of the cobalamin cofactor "(cbl).
Because the number of patients with homocystinuria is low, the disease is considered rare, and Cystadane was designated an 'orphan medicine' (a medicine used in rare diseases) on 9 July 2001.
The medicine can only be obtained with a prescription.
How is Cystadane used?
Treatment with Cystadane should be supervised by a physician experienced in the treatment of patients with homocystinuria.
The standard dose of Cystadane recommended for patients over 10 years of age is 6 g per day, divided into two equal doses. Children under the age of 10 should take 100 mg per kilogram of body weight per day, divided into two equal doses. The dose for children can be adjusted according to the response to treatment (monitored by measuring homocysteine levels in the blood); however, increasing the frequency beyond twice daily or increasing the dose beyond 150 mg per kilogram of body weight does not improve the benefits of Cystadane. The aim of treatment is to keep plasma homocysteine levels below 15 μM or below the value. as low as possible.This is usually achieved within one month.
3 measuring spoons are supplied with Cystadane, delivering 100 mg, 150 mg and 1 g of
dust. The dose should be completely dissolved in water, juice, milk, infant formula or food and should be swallowed immediately after mixing.
How does Cystadane work?
Betaine is a natural substance extracted from sugar beet. In homocystinuria, betaine reduces plasma homocysteine levels by facilitating a reaction in the body called "remethylation", whereby homocysteine is transformed back into methionine, thus improving the symptoms of the disease.
How has Cystadane been studied?
The company presented data on Cystadane from the scientific literature. Among these were 202 reports describing the effects of Cystadane, given at various doses, on homocysteine levels in homocystinuria patients of different ages. Information on symptoms, dose and duration of treatment, and other medications taken was also provided for 140 patients
at the same time. Most patients were also taking vitamin B6 or B12, or folate. The information from these studies was compared with published reports describing the observed outcome in untreated patients with the same disease.
What benefit has Cystadane shown during the studies?
Patients taking Cystadane showed greater reductions in homocysteine levels than untreated patients, associated with improvement in cardiovascular (heart and blood vessel) symptoms and nervous system improvement reported by approximately three-quarters of patients. under treatment with Cystadane. The medicine was effective in treating patients with all three types of homocystinuria.
What is the risk associated with Cystadane?
The most frequent side effects with Cystadane (seen in more than 1 in 10 patients) are levels
elevated plasma levels of methionine. Methionine levels need to be monitored in patients taking Cystadane, as an increase in these levels may be associated with brain edema (swelling of the brain). Patients presenting with symptoms of cerebral edema such as morning headaches with vomiting or visual changes should notify their doctor, as treatment with Cystadane may need to be stopped. For the full list of side effects reported with Cystadane, see the Package Leaflet. Cystadane should not be used in patients who are hypersensitive (allergic) to betaine.
Why has Cystadane been approved?
The Committee for Medicinal Products for Human Use (CHMP) concluded that, despite the limited systematic studies conducted with Cystadane, the medicine is useful as an adjunct therapy in the treatment of homocystinuria, as a supplement to vitamin-based therapies and in combination with a specific diet The committee notes that Cystadane is not a substitute for other treatments.
The CHMP decided that Cystadane's benefits are greater than its risks as an add-on treatment for homocystinuria, when used according to the product's directions, and therefore recommended that it be given a Marketing Authorization.
What measures are being taken to ensure the safe use of Cystadane?
The company that makes Cystadane will set up a registry of patients taking the medicine for
monitor their safety. In particular, it will monitor cases of cerebral edema, which have been observed in a small number of patients during the drug's trial.
Other information about Cystadane:
On February 15, 2007, the European Commission granted Orphan Europe SARL a "Marketing Authorization" for Cystadane, valid throughout the European Union.
To register the designation of Cystadane as an orphan drug click here.
For the full version of the Cystadane EPAR click here.
Last update of this summary: 01-2007.
The information on Cystadane - Betaine Anhydrous published on this page may be out of date or incomplete. For a correct use of this information, see the Disclaimer and useful information page.
