Carbaglu 200 mg dispersible tablets

Carbaglu can help eliminating excess of hyperammonaemia (elevated ammonia level in the blood), due to the lack of a specific liver enzyme N- acetylglutamate synthase. Patients with this rare disorder are not able to eliminate nitrogen waste, which builds up after eating protein. Nitrogen waste is in the form of ammonia, which is especially toxic for the brain and leads, in severe cases, to reduced levels of consciousness and to coma.
This disorder will persist during the entire life time of the patient and therefore the need for this treatment is lifelong.

Do not take Carbaglu:
Do not take Carbaglu if you are hypersensitive (allergic) to carglumic acid or any of the other ingredients of Carbaglu.
Do not take Carbaglu during breast-feeding

Take special care with Carbaglu:
Carbaglu treatment should be initiated under the supervision of a physician experienced in the treatment of metabolic disorders.

Your doctor will test your individual responsiveness to carglumic acid before initiating any long term treatment.
The dose should be adjusted individually in order to maintain normal ammonia plasma levels.

Your doctor may prescribe supplemental arginine or restrict your protein intake.

In order to follow-up your condition and your treatment, your doctor may examine your liver, your kidneys, your heart and your blood on a regular basis.

Taking other medicines
Please tell your doctor or pharmacist if you are taking or have recently taken any other medicines, including medicines obtained without a prescription

Taking Carbaglu with food and drink

Carbaglu must be taken orally before meals or feedings.
The tablets must be dispersed in a minimum of 5 to 10 ml of water and taken immediately. The suspension has a slightly acidic taste.

Pregnancy and Breast-feeding

The effects of Carbaglu on pregnancy and the unborn child are not known. Please consult your doctor for advice if you are pregnant or planning to become pregnant.
The excretion of carglumic acid into breast milk has not been studied in women. Nevertheless, as carglumic acid has been shown to be present in the milk of lactating rats with potential toxic effects for their fed pups, you should not breast feed your baby if you are taking Carbaglu.

Driving and using machines
Effects on the ability to drive and use machines are not known.

Always take Carbaglu exactly as your doctor has instructed you. You should check with your doctor or pharmacist if you are unsure.

The usual dose:

• the initial daily dose is usually 100 mg per kilogram of body weight, up to a maximum of 250 mg per kilogram of body weight (for example, if you weight 10kg, you should take 1g per day, or 5 tablets),
• in the long term, the daily dose usually ranges from 10 mg to 100 mg per kilogram of body weight. Your doctor will determine the dose suitable to you in order to maintain normal ammonia levels in your blood.

When the patient is in hyperammonaemic coma, Carbaglu is administered by fast push through a syringe via the tube set up and used to feed you..

If you take more Carbaglu than you should:
Ask your doctor or pharmacist for advice.

If you forget to take Carbaglu:
Do not take a double dose to make up for forgotten individual doses.

Like all medicines, Carbaglu can have side effects.

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).

• Common: increased sweating
• Uncommon: increased transaminases

If you notice any side effects, please inform your doctor or pharmacist.

Keep out of the reach and sight of children.
Do not use after the expiry date stated on the tablet container.

Store in a refrigerator (2°C ? 8°C)

After first opening of the container: do not refrigerate, do not store above 30°C.
Keep the container tightly closed in order to protect from moisture.
Write the date of opening on the tablet container. Discard 1 month after first opening.

Carbaglu 200 mg dispersible tablets

Carglumic acid

• The active substance is carglumic acid. Each tablet contains 200 mg of carglumic acid.
• The other ingredients are microcrystalline cellulose, sodium laurilsulfate, hypromellose, croscarmellose sodium, silica colloidal anhydrous, sodium stearyl fumarate.

Marketing Authorisation Holder and Manufacturer

Orphan Europe SARL
Immeuble ?Le Wilson?
70 avenue du Général de Gaulle
F-92800 Puteaux
France
Tel: + 33 1 4773 6458
Fax: + 33 1 4900 1800

For any information about this medicinal product, please contact the local representative of the Marketing Authorisation Holder.

BelgiqueBelgiëBelgien Orphan Europe Benelux Koning Albert 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

Magyarország Orphan Europe Germany GmbH Orphan Europe Germany GmbH Max-Planck Str. 6

D-63128 Dietzenbach Te. 49 6074 812160 Max-Planck Str. 6 D - 63128 Dietzenbach Németország Tel 49 6074 812160

eská republika Orphan Europe Germany GmbH Max-Planck Str. 6 D - 63128 Dietzenbach Nmecko Tel 49 6074 812160 Malta Orphan Europe SARL Immeuble Le Wilson 70 avenue du Général de Gaulle F-92800 Puteaux Franza Tél 33 1 47 73 64 58

Danmark Orphan Europe AB Banérgatan 37 S 115 22 Stockholm Sverige Tlf 46 8 545 80 230 Nederland Orphan Europe Benelux Koning Albert I laan 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 812160 Norge Orphan Europe AB Banérgatan 37 S 115 22 Stockholm Sverige Tlf 46 8 545 80 230 Eesti Orphan Europe AB Banérgatan 37 S 115 22 Stockholm Rootsi Tel 46 8 545 80 230 Österreich Orphan Europe Germany GmbH Max-Planck Str. 6 D - 63128 Dietzenbach Deutschland Tel 49 6074 812160

Polska Orphan Europe Germany GmbH Max-Planck Str. 6 D - 63128 Dietzenbach Niemcy Tel 49 6074 812160 Orphan Europe SARL Immeuble Le Wilson 70 avenue du Général de Gaulle F-92800 Puteaux Tél 33 1 47 73 64 58 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 70 avenue du Général de Gaulle F-92800 Puteaux Tél 33 01 47 73 64 58 România Orphan Europe Germany GmbH Max-Planck Str. 6 D-63128 Dietzenbach Germania Tel 49 6074 812160

Ireland Orphan Europe UK Ltd. Isis House, 43 Station Road Henley-on-Thames Oxfordshire RG9 1AT, UK Tel 44 1491 414333 Slovenija Orphan Europe Germany GmbH Max-Planck Str. 6 D - 63128 Dietzenbach Nemija Tel 49 6074 812160

Ísland Orphan Europe AB Banérgatan 37 S 115 22 Stockholm Svíjóð Tel 46 8 545 80 230 Slovenská republika Orphan Europe Germany GmbH Max-Planck Str. 6 D - 63128 Dietzenbach Nemecko Tel 49 6074 812160

Italia Orphan Europe Italy Srl Via Cellini 11 I - 20090 Segrate Milano Tel 39 02 26 95 01 39 SuomiFinland Orphan Europe AB Banérgatan 37 S 115 22 Stockholm Sverige PuhTel 46 8 545 80 230

Sverige Orphan Europe AB Banérgatan 37 S 115 22 Stockholm Tel 46 08 545 80 230 Orphan Europe SARL Immeuble Le Wilson 70 avenue du Général de Gaulle F-92800 Puteaux Tél 33 1 47 73 64 58

Latvija Orphan Europe AB Banérgatan 37 S 115 22 Stockholm Zviedrija Tel 46 8 545 80 230 United Kingdom Orphan Europe UK Ltd. Isis House, 43 Station Road Henley-on-Thames Oxfordshire RG9 1AT Tel 44 01491 414333

Lietuva
Orphan Europe AB
Banérgatan 37
S ? 115 22 Stockholm
?vedija
Tel: +46 8 545 80 230

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