Страна: Европейски съюз
Език: английски
Източник: EMA (European Medicines Agency)
galsulfase
BioMarin International Limited
A16AB
galsulfase
Other alimentary tract and metabolism products,
Mucopolysaccharidosis VI
Naglazyme is indicated for long-term enzyme-replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis VI (MPS VI; N-acetylgalactosamine-4-sulfatase deficiency; Maroteaux-Lamy syndrome) (see section 5.1). As for all lysosomal genetic disorders, it is of primary importance, especially in severe forms, to initiate treatment as early as possible, before appearance of non-reversible clinical manifestations of the disease. A key issue is to treat young patients aged
Revision: 19
Authorised
2006-01-23
19 B. PACKAGE LEAFLET 20 PACKAGE LEAFLET: INFORMATION FOR THE USER NAGLAZYME 1 MG/ML CONCENTRATE FOR SOLUTION FOR INFUSION Galsulfase READ ALL OF THIS LEAFLET CAREFULLY BEFORE YOU START USING 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. - If any of the side effects gets serious, or if you notice any side effects not listed in this leaflet, please tell your doctor. WHAT IS IN THIS LEAFLET : 1. What this medicine is and what it is used for 2. What you need to know before you are given this medicine 3. How this medicine is given 4. Possible side effects 5. How to store this medicine 6. Contents of the pack and other information 1. WHAT THIS MEDICINE IS AND WHAT IT IS USED FOR Naglazyme is used to treat patients with MPS VI disease (Mucopolysaccharidosis VI). People with MPS VI disease have either a low level, or no level, of an enzyme called N-acetylgalactosamine 4-sulfatase, which breaks down specific substances (glycosaminoglycans) in the body. As a result, these substances do not get broken down and processed by the body as they should. They accumulate in many tissues in the body, which causes the symptoms of MPS VI. HOW THIS MEDICINE WORKS This medicine contains a recombinant enzyme called galsulfase. This can replace the natural enzyme which is lacking in MPS VI patients. Treatment has been shown to improve walking and stair- climbing ability, and to reduce the levels of glycosaminoglycans in the body. This medicine may improve the symptoms of MPS VI. 2. WHAT YOU NEED TO KNOW BEFORE YOU ARE GIVEN THIS MEDICINE YOU MUST NOT RECEIVE THIS MEDICINE - If you have experienced severe or life-threatening allergic (hypersensitive) reactions to galsulfase or any of the other ingredients of Naglazyme and re-administration of the medicine was not successful. WARNINGS AND PRECAUTIONS - If you are treated with Naglazyme, you may develop infusion-associated reactions. An infusion associated reaction is Прочетете целия документ
1 _ _ ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS 2 1. NAME OF THE MEDICINAL PRODUCT Naglazyme 1 mg/ml concentrate for solution for infusion 2. QUALITATIVE AND QUANTITATIVE COMPOSITION Each ml of solution contains 1 mg galsulfase. One vial of 5 ml contains 5 mg galsulfase. Galsulfase is a recombinant form of human N-acetylgalactosamine 4-sulfatase and is produced by recombinant DNA technology using mammalian Chinese Hamster Ovary (CHO) cell culture. _Excipients _ Each 5 ml vial contains 0.8 mmol (18.4 mg) of sodium. For a full list of excipients, see section 6.1. 3. PHARMACEUTICAL FORM Concentrate for solution for infusion. A clear to slightly opalescent, and colourless to pale yellow solution. 4. CLINICAL PARTICULARS 4.1 THERAPEUTIC INDICATIONS Naglazyme is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Mucopolysaccharidosis VI (MPS VI; N-acetylgalactosamine 4-sulfatase deficiency; Maroteaux-Lamy syndrome) (see section 5.1). 4.2 POSOLOGY AND METHOD OF ADMINISTRATION As for all lysosomal genetic disorders, it is of primary importance, especially in severe forms, to initiate treatment as early as possible, before appearance of non-reversible clinical manifestations of the disease. Naglazyme treatment should be supervised by a physician experienced in the management of patients with MPS VI or other inherited metabolic diseases. Administration of Naglazyme should be carried out in an appropriate clinical setting where resuscitation equipment to manage medical emergencies would be readily available _. _ _ _ Posology The recommended dose regimen for galsulfase is 1 mg/kg body weight administered once every week as an intravenous infusion over 4 hours. _Special populations _ _ _ _Elderly _ The safety and efficacy of Naglazyme in patients older than 65 years has not been established, and no alternative dose regimen can be recommended in these patients. _Renal and hepatic impairment _ 3 The safety and efficacy of Naglazyme in patients with renal or hepatic insufficiency have n Прочетете целия документ