Country: Evrópusambandið
Tungumál: enska
Heimild: EMA (European Medicines Agency)
carglumic acid
Recordati Rare Diseases
A16AA05
carglumic acid
Other alimentary tract and metabolism products,
Amino Acid Metabolism, Inborn Errors; Propionic Acidemia
Carbaglu is indicated in treatment of:hyperammonaemia due to N-acetylglutamate-synthase primary deficiency;hyperammonaemia due to isovaleric acidaemia;hyperammonaemia due to methymalonic acidaemia;hyperammonaemia due to propionic acidaemia.
Revision: 19
Authorised
2003-01-24
19 B. PACKAGE LEAFLET 20 PACKAGE LEAFLET: INFORMATION FOR THE USER CARBAGLU 200 MG DISPESIBLE TABLETS carglumic acid READ ALL OF THIS LEAFLET CAREFULLY BEFORE YOU START TAKING THIS MEDICINE BECAUSE IT CONTAINS IMPORTANT INFORMATION FOR YOU. - Keep this leaflet. You may need to read it again. - If you have any further questions, ask your doctor or pharmacist. - This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, even if their signs of illness are the same as yours. - If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects not listed in this leaflet. See section 4. WHAT IS IN THIS LEAFLET : 1. What Carbaglu is and what it is used for 2. What you need to know before you take Carbaglu 3. How to take Carbaglu 4. Possible side effects 5 How to store Carbaglu 6. Contents of the pack and other information 1. WHAT CARBAGLU IS AND WHAT IT IS USED FOR Carbaglu can help eliminating excessive ammonia plasma levels (elevated ammonia level in the blood). Ammonia is especially toxic for the brain and leads, in severe cases, to reduced levels of consciousness and to coma. Hyperammonaemia may be 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. This disorder persists during the entire life of the affected patient and therefore the need for this treatment is lifelong. • isovaleric acidaemia, methylmalonic acidaemia or propionic acidaemia. Patients suffering from one of these disorders need treatment during the hyperammonaemia crisis. 2. WHAT YOU NEED TO KNOW BEFORE YOU TAKE CARBAGLU DO NOT TAKE CARBAGLU if you are allergic to carglumic acid or any of the other ingredients of Carbaglu (listed in section 6). Do not take Carbaglu during breast-feeding WARNINGS AND PRECAUTIONS Talk to your doctor or pharmacist before taking Carbaglu. Carbaglu treatment should be initiated under the supervision of a physician Lestu allt skjalið
1 ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS _ _ 2 1. NAME OF THE MEDICINAL PRODUCT Carbaglu 200 mg dispersible tablets 2. QUALITATIVE AND QUANTITATIVE COMPOSITION Each tablet contains 200 mg of carglumic acid. For a full list of excipients, see section 6.1 3. PHARMACEUTICAL FORM Dispersible tablet The tablets are white and elongated with three score marks and engraved on one side. The tablet can be divided into equal doses. 4. CLINICAL PARTICULARS 4.1 THERAPEUTIC INDICATIONS Carbaglu is indicated in treatment of • hyperammonaemia due to N-acetylglutamate synthase primary deficiency. • hyperammonaemia due to isovaleric acidaemia. • hyperammonaemia due to methylmalonic acidaemia. • hyperammonaemia due to propionic acidaemia. 4.2 POSOLOGY AND METHOD OF ADMINISTRATION Carbaglu treatment should be initiated under the supervision of a physician experienced in the treatment of metabolic disorders. Posology: • For N-acetylglutamate synthase deficiency: Based on clinical experience, the treatment may be started as early as the first day of life. The initial daily dose should be 100 mg/kg, up to 250 mg/kg if necessary. It should then be adjusted individually in order to maintain normal ammonia plasma levels (see section 4.4). In the long term, it may not be necessary to increase the dose according to body weight as long as adequate metabolic control is achieved; daily doses range from 10 mg/kg to 100 mg/kg. _Carglumic acid responsiveness test _ It is recommended to test individual responsiveness to carglumic acid before initiating any long term treatment. As examples - In a comatose child, start with a dose of 100 to 250 mg/kg/day and measure ammonia plasma concentration at least before each administration; it should normalise within a few hours after starting Carbaglu. - In a patient with moderate hyperammonaemia, administer a test dose of 100 to 200 mg/kg/day for 3 days with a constant protein intake and perform repeated determinations of ammonia plasma concentration (before and 1 hour after a meal); adjust th Lestu allt skjalið