CYSTAGON 50 mg hard capsules

Cystinosis is a metabolic disease called ?nephropathic cystinosis? which is characterized by an abnormal accumulation of the amino acid cystine in various organs of the body such as the kidney, eye, muscle, pancreas, and brain. Cystine build up causes kidney damage and excretion of excess amounts of glucose, proteins and electrolytes. Different organs are affected at different ages.

CYSTAGON is prescribed to manage this rare inherited disorder. CYSTAGON is a medicine that reacts with cystine to decrease its level within the cells.

Do not use CYSTAGON

• if you -or your child- are allergic (hypersensitive) to cysteamine bitartrate or penicillamine or any of the other ingredients of Cystagon.
• If you are pregnant, this is particularly relevant during the first trimester
• if you are breast-feeding.

Take special care with CYSTAGON

• When your or your child?s disorder has been confirmed by leucocyte cystine measurements, the therapy with CYSTAGON must be started as soon as possible.
• A few cases of skin lesions on elbows like little hard lumps have been reported in children treated with high doses of different cysteamine preparations. These lesions were associated with skin striae and bone lesions such as fracture and bone deformities, and with laxity of joints. Your doctor could require a regular physical and X-ray examination for the skin and the bones to control the effects of the medicinal product. Self examination of your or your child?s skin is recommended. If any skin or bone abnormalities appear, please inform your doctor immediately.
• Your doctor could require controlling the blood cell count on a regular basis.
• CYSTAGON has not been shown to prevent cystine crystals accumulating in the eye. Where cysteamine ophthalmic solution has been used for that purpose, its usage should continue.
• In contrast to phosphocysteamine, another active substance close to cysteamine bitartrate, CYSTAGON does not contain phosphate. You may already be receiving phosphate supplements

and the dose of these may need to be changed when CYSTAGON is substituted for phosphocysteamine.

• To avoid any risk of aspiration in the lungs, the capsules should not be given to children under the age of approximately 6 years.

Using other medicines

Please inform your doctor or pharmacist if you are taking or have recently taken any other medicines, even those not prescribed.

Using CYSTAGON with food and drink
For children under approximately six years of age, the hard capsule may be opened and the contents sprinkled on food (e.g. milk, potatoes or starch based foods) or mixed in formula. Do not add to acidic drinks e.g. orange juice. Consult the doctor for complete directions.

Pregnancy
You should not use CYSTAGON if you are pregnant. Please consult your doctor if you plan to become pregnant.

Breast-feeding

CYSTAGON must not be used during breast-feeding.

Driving and using machines

CYSTAGON may cause some drowsiness. When starting therapy, you or your child should not engage in potentially hazardous activities until the effects of the drug are well known.

Always use CYSTAGON exactly as your doctor or your child?s doctor has told you. You should check with your doctor if you are not sure.
The dose of CYSTAGON prescribed for you or your child will depend on your or your child's age and weight.
For children up to age 12 years, the dose will be based on the body size (surface area), the usual dose

being 1.30 g/m 2 of body surface area per day.
For patients over age 12 and over 50 kg weight, the usual dose is 2g/day.
In any case the usual dose should not exceed 1.95 g/m 2/day.
CYSTAGON should be taken or given only by mouth and exactly as your or your child's doctor directs. In order for CYSTAGON to work correctly, you must do the following:

• Follow your doctor's directions exactly. Do not increase or decrease the amount of medicine without your doctor's approval.
• Hard capsules should not be given to children under approximately six years of age because they may not be able to swallow them and they may choke. For children under approximately six years of age, the hard capsule may be opened and the contents sprinkled on food (e.g. milk, potatoes or starch based foods) or mixed in formula. Do not add to acidic drinks e.g. orange juice. Consult the doctor for complete directions.
• Your or your child's medical treatment may include, in addition to CYSTAGON, one or more supplements to replace important electrolytes lost through the kidneys. It is important to take or give these supplements exactly as instructed. If several doses of the supplements are missed or weakness or drowsiness develops, call the doctor for instructions.
• Regular blood tests to measure the amount of cystine inside white blood cells are necessary to help determine the correct dose of CYSTAGON. Your or your child's doctor will arrange for the blood tests to be done. Regular blood and urine tests to measure the levels of the body's important electrolytes are also necessary to help your or your child's doctor correctly adjust the doses of these supplements.

CYSTAGON should be taken 4 times a day, every 6 hours, preferably just after or with food. It is important to take the dose as close to every 6 hours as possible.

Treatment with CYSTAGON should continue indefinitely, as instructed by your doctor.

If you use more CYSTAGON than you should:

You should contact your or your child's doctor or the hospital emergency department immediately if more medicine has been taken than has been prescribed, drowsiness develops .

If you forget to take CYSTAGON:

If a dose of medicine is missed, it should be taken as soon as possible. However if it is within two hours of the next dose, skip the missed dose and go back to the regular dosing schedule. Do not take a double dose to make up for a forgotten dose.

Like all medicines, CYSTAGON can cause side effects, although not everybody gets them.

CYSTAGON may cause some people to become drowsy or less vigilant than they are normally. Make sure you know how you or your child reacts to this medicine before doing anything that could be dangerous if not alert.

The following side effects were reported as follows: very common (occurring in at least one in 10 patients), common (occurring in at least one in 100 patients), uncommon (occurring in at least one in1,000 patients), rare (occurring in at least one in 10,000 patients), very rare (occurring in at least one in 100,000 patients).

• Very common: vomiting, nausea, diarrhoea, loss of appetite, fever and sensation of sleep
• Common :abdominal pain or discomfort, unpleasant breath and body odour, skin eruption, gastroenteritis, fatigue, headache, encephalopathy (brain disorder) and liver function test abnormalities.
• Uncommon: skin striae, skin lesion (little-hard lumps on elbows), joint laxity, leg pain, bone fracture, scoliosis (deviation of the vertebral column), bone deformity and fragility, hair discoloration, severe allergic reaction, somnolence, fits, nervousness, hallucination, decrease in white blood cells, gastrointestinal ulcer manifested by bleeding in the digestive tract and effect on the kidney manifested by swelling of the extremities and weight gain . Since some of these side effects are serious, ask your or your child?s doctor to explain their warning signs.

If any of the side effects gets serious, or if you notice any other side effects not listed in this leaflet, please tell your doctor or pharmacist.

Keep out of the reach and sight of children.

Do not use after the expiry date which is stated on the label. The expiry date refers to the last day of that month.

Do not store above 25°C and keep the container tightly closed in order to protect from light and moisture.

What CYSTAGON contains

• The active substance is cysteamine bitartrate (mercaptamine bitartrate). Each hard capsule of CYSTAGON 50 mg contains 50 mg of cysteamine (as mercaptamine bitartrate) Each hard capsule of CYSTAGON 150 mg contains 150 mg of cysteamine (as mercaptamine bitartrate)
• The other ingredients are microcrystalline cellulose, starch, pregelatinized, magnesium stearate/sodium lauryl sulphate, colloidal silicon dioxide, croscarmellose sodium, capsule shells: gelatin, titanium dioxide, black ink on hard capsules (E172).

What CYSTAGON looks like and contents of the pack

Hard Capsules

• Cystagon 50 mg: white, opaque hard capsules with CYSTA 50 on the body and MYLAN on the cap. Bottles of 100 or 500 hard capsules. All pack sizes may be not marketed.
• Cystagon 150 mg: white, opaque hard capsules with CYSTAGON 150 on the body and MYLAN on the cap. Bottles of 100 or 500 hard capsules. All pack sizes may be not marketed.

Marketing Authorisation Holder and Manufacturer: Orphan Europe SARL, Immeuble ?Le Wilson?, F-92058 Paris La Défense, France

For any information about this medicine, please contact the local representative of the Marketing Authorisation Holder:

BelgiqueBelgiëBelgien Orphan Europe Benelux Koning Albert I laan 48 bus 3 BE-1780 Wemmel Brussels Tel 32 2 46101 36 LuxembourgLuxemburg Orphan Europe Benelux Koning Albert I laan 48 bus 3 BE-1780 WemmelBrussels BelgiqueBelgien Tel 32 2 46101 36

Orphan Europe Germany GmbH Max-Planck Str. 6 D-63128 Dietzenbach Te. 49 6074 914090 Magyarország Orphan Europe Germany GmbH Max-Planck Str. 6 D-63128 Dietzenbach Németország Tel. 49 6074 914090

eská republika Orphan Europe Germany GmbH Max-Planck Str. 6 D-63128 Dietzenbach Nmecko Tel 49 6074 914090 Malta Orphan Europe SARL Immeuble Le Wilson F-92058 Paris La Défense Franza Tel 33 1 47 73 64 58

Danmark Swedish Orphan AS Wilders Plads 5 DK - 1403 København K Tlf 45 32 96 68 69 Nederland Orphan Europe Benelux Koning Albert I Iaan 48 bus 3 BE-1780 Wemmel Brussels België Tel 32 2 46101 36

Deutschland Orphan Europe Germany GmbH Max-Planck Str. 6 D-63128 Dietzenbach Tel 49 06074 914090 Norge Swedish Orphan AS Trollåsveien 6 N 1414 Trollåsen Tlf 47 66 82 34 00

Eesti Orphan Europe AB Banérgatan 37 S-11522 Stockolm Rootsi Tel 46 8 545 80 230 Österreich Orphan Europe Germany GmbH Max-Planck Str. 6 D-63128 Dietzenbach Deutschland Tel 49 6074 914090

Orphan Europe SARL Immeuble Le Wilson F-92058 Paris La Défense 33 1 47 73 64 58 Polska Orphan Europe Germany GmbH Max-Planck Str. 6 D-63128 Dietzenbach Niemcy Tel. 49 6074 914090

España Orphan Europe, S.L. Gran via de les Cortes Catalanes, 649 Despacho, n1 E-08010 Barcelona Tel 34 93 342 51 20 Portugal Orphan Europe, S.L. Gran via de les Cortes Catalanes, 649 Despacho, n1 E-08010 Barcelona Espanha Tel 34 93 342 51 20

France Orphan Europe SARL Immeuble Le Wilson F-92058 Paris La Défense Tél 33 01 47 73 64 58 România Orphan Europe Germany GmbH Max-Planck Str. 6 D-63128 Dietzenbach Germania Tel 49 6074 914090

Slovenija Orphan Europe Germany GmbH Max-Planck Str. 6 D-63128 Dietzenbach Nemija Tel 49 6074 914090 Ireland Orphan Europe UK Ltd. Isis House, 43 Station road Henley-on-Thames Oxfordshire RG9 1AT, UK United Kingdom Tel 44 1491 414333

Ísland Swedish Orphan AS co Ísfarm ehf. Lynghálsi 13 IS 110 Reykjavik Tel 354 540 8080 Slovenská republika Orphan Europe Germany GmbH Max-Planck Str. 6 D-63128 Dietzenbach Nemecko Tel 49 6074 914090

Italia Orphan Europe Italy Srl Via Cellini 11 I-20090 Segrate Milano Tel 39 02 26 95 01 39 SuomiFinland Oy Swedish Orphan Ab Rajatorpantie 41 C FIN - 01640 Vantaa PuhTfn 358 09 8520 2150

Sverige Swedish Orphan AB Drottninggatan 98 S - 111 60 Stockholm Tfn 46 08 412 98 00 Orphan Europe SARL Immeuble Le Wilson F - 92058 Paris La Défense 33 1 47 73 64 58

Latvija Orphan Europe AB Banérgatan 37 S-11522 Stockolm Zviedrija Tel 46 8 545 80 230 United Kingdom Orphan Europe UK Ltd. Isis House, 43 Station road Henley-on-Thames Oxfordshire RG9 1AT, UK Tel 44 01491 414333

Lietuva
Orphan Europe AB
Banérgatan 37
S-11522 Stockolm
?vedija
Tel: + 46 8 545 80 230

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