Kraj: Unia Europejska
Język: angielski
Źródło: EMA (European Medicines Agency)
glycerol phenylbutyrate
Immedica Pharma AB
A16AX09
glycerol phenylbutyrate
Other alimentary tract and metabolism products,
Urea Cycle Disorders, Inborn
Ravicti is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) including deficiencies of carbamoyl phosphate-synthase-I (CPS), ornithine carbamoyltransferase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL), arginase I (ARG) and ornithine translocase deficiency hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome (HHH) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.Ravicti must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).
Revision: 16
Authorised
2015-11-26
26 B. PACKAGE LEAFLET 27 PACKAGE LEAFLET: INFORMATION FOR THE PATIENT RAVICTI 1.1 G/ML ORAL LIQUID glycerol phenylbutyrate 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 RAVICTI is and what it is used for 2. What you need to know before you take RAVICTI 3. How to take RAVICTI 4. Possible side effects 5. How to store RAVICTI 6. Contents of the pack and other information 1. WHAT RAVICTI IS AND WHAT IT IS USED FOR RAVICTI contains the active substance ‘glycerol phenylbutyrate’ which is used to treat six known ‘urea cycle disorders’ (UCDs) in adults and children. The UCDs include deficiencies of certain liver enzymes such as carbamoyl phosphate synthetase I (CPS), ornithine carbamoyltransferase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL), arginase I (ARG) and ornithine translocase deficiency hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome (HHH). RAVICTI must be combined with a diet reduced in protein intake, and in some cases a diet with supplements such as essential amino acids (arginine, citrulline, protein-free calorie supplements). ABOUT UREA CYCLE DISORDERS • In urea cycle disorders, the body cannot remove the nitrogen from the protein that we eat. • Normally, the body turns the extra nitrogen in the protein into a waste compound called ‘ammonia’. The liver then removes ammonia from the body through a cycle called the ‘urea cycle’. • In urea cycle disorders, the body is not able to produce enough liver enzymes to remove Przeczytaj cały dokument
1 ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS _ _ _ _ 2 1. NAME OF THE MEDICINAL PRODUCT RAVICTI 1.1 g/ml oral liquid 2. QUALITATIVE AND QUANTITATIVE COMPOSITION Each ml of liquid contains 1.1 g of glycerol phenylbutyrate. This corresponds to a density of 1.1 g/ml. 3. PHARMACEUTICAL FORM Oral liquid. Clear, colourless to pale yellow liquid. 4. CLINICAL PARTICULARS 4.1 THERAPEUTIC INDICATIONS RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) including deficiencies of carbamoyl phosphate synthetase I (CPS), ornithine carbamoyltransferase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL), arginase I (ARG) and ornithine translocase deficiency hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome (HHH) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). 4.2 POSOLOGY AND METHOD OF ADMINISTRATION RAVICTI should be prescribed by a physician experienced in the management of UCDs. Posology RAVICTI must be used with dietary protein restriction and sometimes dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements) depending on the daily dietary protein intake needed to promote growth and development. The daily dose should be individually adjusted according to the patient’s protein tolerance and the daily dietary protein intake needed. RAVICTI therapy may be required life long unless orthotopic liver transplantation is elected. _Adults and children _ The recommended dose for patients naïve to phenylbutyric acid and for patients switching from sodium phenylbutyrate or from sodium phenylacetate/sodium benzoate injection to RAVICTI are different. The recommended total daily dose of RAVICTI is based on body surface area and ranges from 4.5 ml/m 2 /day to 11.2 ml/ Przeczytaj cały dokument