Prescription Information for the Patient

Cystadane 1 g Oral Powder

Anhydrous Betaine

Read this entire prescription carefully before starting to take the medication as it contains important information for you.

• Keep this prescription, as you may need to refer to it again.
• If you have any questions, consult your doctor or pharmacist.
• This medication has been prescribed only for you. Do not give it to others, even if they have the same symptoms as you, as it may harm them.
• If you experience any adverse effects, consult your doctor or pharmacist, even if they are not listed in this prescription. See section 4.

Cystadane contains anhydrous betaine, which is indicated for the supplementary treatment of homocystinuria, a hereditary (genetic) disease in which the body is unable to completely break down the amino acid methionine.

Methionine is present in the proteins of normal foods (e.g.: meat, fish, milk, cheese, eggs). It is converted into homocysteine, which is then usually converted into cysteine during digestion. Homocystinuria is a disease caused by the accumulation of homocysteine that does not convert into cysteine and is characterized by the formation of blood clots in the veins, bone weakness, and skeletal and crystalline anomalies. The use of Cystadane along with other treatments such as vitamin B6, vitamin B12, folate, and a specific diet aims to reduce elevated levels of homocysteine in the body.

Do not take Cystadane

If you are allergic to anhydrous betaine.

Warnings and precautions

Consult your doctor or pharmacist before starting to take Cystadane.

If you experience side effects such as headaches, vomiting, or changes in vision and have the subtype of homocystinuria known as CBS (cystathionine beta-synthase deficiency), contact your doctor immediately, as they may be signs of brain mass inflammation (cerebral edema). In this case, your doctor will monitor your methionine level in your body and may review your diet. It may be necessary to interrupt treatment with Cystadane.

If you are receiving treatment with Cystadane and an amino acid mixture, and if you need to take other medications at the same time, leave a 30-minute gap between doses (see section “Other medications and Cystadane”).

Other medications and Cystadane

Inform your doctor if you are taking, have recently taken, or may need to take any other medication.

If you are using an amino acid mixture or medications such as vigabatrin or GABA analogs (medication used to treat epilepsy), please inform your doctor as they may interact with your Cystadane treatment.

Pregnancy and breastfeeding

If you are pregnant or breastfeeding, think you may be pregnant, or intend to become pregnant, consult your doctor before using this medication. Your doctor will decide if you can use the medication during pregnancy and breastfeeding.

Driving and operating machines

The influence of Cystadane on the ability to drive and operate machines is negligible or insignificant.

This medication's use will be monitored by a doctor experienced in treating patients with homocystinuria.

Follow exactly the administration instructions for this medication as indicated by your doctor or pharmacist. If in doubt, consult your doctor or pharmacist again.

The recommended dose in children and adults is 100 mg/kg/day in two doses per day. In some cases, doses higher than 200 mg/kg/day may be necessary to achieve therapeutic goals. Your doctor may adjust the dose based on laboratory values.

You will need to have blood tests performed periodically to determine the correct daily dose.

You must take Cystadane orally (by mouth).

To measure the dose:

• shake the bottle lightly before opening it

• take the appropriate measuring spoon:
• the small spoon provides 100 mg of anhydrous betaine powder;
• the medium spoon provides 150 mg of anhydrous betaine powder;
• the large spoon provides 1 g of anhydrous betaine powder.
• remove a filled spoon of powder from the bottle
• scrape the spoon with the flat side of a knife
• the powder left in the spoon is a spoonful
• remove the correct number of spoonfuls of powder from the bottle

Mix the measured dose of powder with water, juice, milk, infant formula powder, or food until it dissolves completely and take it immediately after mixing.

If you take more Cystadane than you should

If you accidentally take an excessive amount of Cystadane, inform your doctor or pharmacist immediately.

If you forget to take Cystadane:

Do not take a double dose to make up for missed doses. If you forget to take a dose, take it as soon as you remember and continue with the next dose as scheduled.

If you interrupt treatment with Cystadane

Do not stop treatment without consulting your doctor. Consult your doctor or pharmacist before stopping treatment.

If you have any other questions about using this medication, ask your doctor or pharmacist.

Like all medications, this medication may produce adverse effects, although not all people will experience them.

The most frequent adverse effect of taking Cystadane, which may affect more than 1 in 10 people (very frequent), is the presence of elevated levels of methionine in the blood.

The level of methionine may be related to cerebral edema (brain swelling), which may affect up to 1 in 100 people (infrequent). If you experience morning headaches with vomiting and/or visual disturbances, consult your doctor immediately (these may be signs of brain swelling).

Gastrointestinal disorders such as diarrhea, nausea, vomiting, stomach discomfort, and tongue inflammation may appear infrequently (may affect up to 1 in 100 people).

Other infrequent adverse effects (may affect up to 1 in 100 people) may include decreased appetite (anorexia), agitation, irritability, hair loss, urticaria, abnormal skin odor, and incontinence of urine.

Reporting Adverse Effects

If you experience any type of adverse effect, consult your doctor or pharmacist, even if it is a possible adverse effect not listed in this prospectus. You can also report them directly through the national notification system included in Appendix V. By reporting adverse effects, you can contribute to providing more information about the safety of this medication.

Keep this medication out of the sight and reach of children.

Do not use this medication after the expiration date that appears on the label of the bottle and box after CAD. The expiration date is the last day of the month indicated.

Do not store above 25°C.

Keep the bottle perfectly closed to protect it from moisture.

Once the bottle is opened, the medication must be used within 3 months.

Medicines should not be thrown down the drains or in the trash. Ask your pharmacist how to dispose of the containers and medications that you no longer need. In this way, you will help protect the environment.

Composition of Cystadane

• The active ingredient is anhydrous betaine. 1 g of oral powder contains 1 g of anhydrous betaine.
• There are no other components.

Appearance of Cystadane and contents of the container

Cystadane is a white, crystalline, free-flowing powder. It is presented in child-resistant containers. Each container contains 180 g of powder. Each box contains one container and three graduated spoons.

Marketing Authorization Holder

Recordati Rare Diseases

Immeuble “Le Wilson”

70, Avenue du General de Gaulle

F-92 800 Puteaux

France

Responsible for manufacturing

Recordati Rare Diseases

Immeuble “Le Wilson”

70, Avenue du General de Gaulle

F-92 800 Puteaux

France

or

Recordati Rare Diseases

Eco River Parc

30, rue des Peupliers

F-92000 Nanterre

France

You can request more information about this medicine by contacting the local representative of the marketing authorization holder.

Last review date of this leaflet

The detailed information about this medicine is available on the website of the European Medicines Agency http://www.emea.europa.eu/. There are also links to other websites about rare diseases and orphan medicines.