Šalis: Europos Sąjunga
kalba: anglų
Šaltinis: EMA (European Medicines Agency)
recombinant human n-acetylgalactosamine-6-sulfatase
BioMarin International Limited
A16AB12
elosulfase alfa
Other alimentary tract and metabolism products,
Mucopolysaccharidosis IV
Vimizim is indicated for the treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA) in patients of all ages.
Revision: 13
Authorised
2014-04-27
20 B. PACKAGE LEAFLET 21 PACKAGE LEAFLET: INFORMATION FOR THE USER VIMIZIM 1 MG/ML CONCENTRATE FOR SOLUTION FOR INFUSION elosulfase alfa ▼This medicine is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you may get. See the end of section 4 for how to report side effects. 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 you get any side effects, talk to your doctor. This includes any possible side effects not listed in this leaflet. See section 4. WHAT IS IN THIS LEAFLET 1. What Vimizim is and what it is used for 2. What you need to know before you are given Vimizim 3. How Vimizim is given 4. Possible side effects 5. How to store Vimizim 6. Contents of the pack and other information 1. WHAT VIMIZIM IS AND WHAT IT IS USED FOR Vimizim contains an enzyme called elosulfase alfa, which belongs to a group of medicines known as enzyme replacement therapies. It is used to treat adults and children with mucopolysaccharidosis type IVA (MPS IVA disease, also known as Morquio A Syndrome). People with MPS IVA disease either lack completely or do not have enough N-acetylgalactosamine-6- sulfatase, an enzyme which breaks down specific substances in the body such as keratan sulphate, which are found in many tissues of the body, including cartilage and bone. As a result, these substances do not get broken down and processed by the body as they should. They accumulate in the tissues interfering with their normal function and causing the symptoms of MPS IVA, such as difficulty walking, trouble breathing, short height, and hearing loss. HOW VIMIZIM WORKS This medicine replaces the natural enzyme N-acetylgalactosamine-6-sulfatase which is lacking in MPS IVA patients. Treatment has been shown to improve walking and to reduce the levels of keratan sulphate in t Perskaitykite visą dokumentą
1 ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS 2 ▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. See section 4.8 for how to report adverse reactions. 1. NAME OF THE MEDICINAL PRODUCT Vimizim 1 mg/ml concentrate for solution for infusion 2. QUALITATIVE AND QUANTITATIVE COMPOSITION Each ml of solution contains 1 mg elosulfase alfa*. Each vial of 5 ml contains 5 mg elosulfase alfa. *Elosulfase alfa is a recombinant form of human N-acetylgalactosamine-6-sulfatase (rhGALNS) and is produced in Chinese Hamster Ovary cell culture by recombinant DNA technology. Excipients with known effect: Each 5 ml vial contains 8 mg sodium and 100 mg sorbitol (E420). For the full list of excipients, see section 6.1. 3. PHARMACEUTICAL FORM Concentrate for solution for infusion (Sterile concentrate). A clear to slightly opalescent and colourless to pale yellow solution. 4. CLINICAL PARTICULARS 4.1 THERAPEUTIC INDICATIONS Vimizim is indicated for the treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA) in patients of all ages. 4.2 POSOLOGY AND METHOD OF ADMINISTRATION Treatment should be supervised by a physician experienced in the management of patients with MPS IVA or other inherited metabolic diseases. Administration of Vimizim should be carried out by an appropriately trained healthcare professional with the ability to manage medical emergencies. Home administration under the supervision of an appropriately trained healthcare professional may be considered for patients who are tolerating their infusions well. _ _ Posology The recommended dose of elosulfase alfa is 2 mg/kg of body weight administered once a week. The total volume of the infusion should be delivered over approximately 4 hours (see Table 1). Because of the potential for hypersensitivity reactions with elosulfase alfa, patients should receive antihistamines with or without antipyretics 30 to 60 min Perskaitykite visą dokumentą