Страна: Европейски съюз
Език: английски
Източник: EMA (European Medicines Agency)
sebelipase alfa
Alexion Europe SAS
A16
sebelipase alfa
Other alimentary tract and metabolism products,
Lipid Metabolism, Inborn Errors
Kanuma is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) deficiency.
Revision: 10
Authorised
2015-08-28
27 B. PACKAGE LEAFLET 28 PACKAGE LEAFLET: INFORMATION FOR THE USER KANUMA 2 MG/ML CONCENTRATE FOR SOLUTION FOR INFUSION sebelipase 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 or your child may get. See the end of section 4 for how to report side effects. READ ALL OF THIS LEAFLET CAREFULLY BEFORE YOU ARE GIVEN 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 KANUMA is and what it is used for 2. What you need to know before you are given KANUMA 3. How KANUMA is given 4. Possible side effects 5. How to store KANUMA 6. Contents of the pack and other information 1. WHAT KANUMA IS AND WHAT IT IS USED FOR KANUMA contains the active substance sebelipase alfa. Sebelipase alfa is similar to the naturally occurring enzyme lysosomal acid lipase (LAL), which the body uses to breakdown fats. It is used to treat patients of all ages with lysosomal acid lipase deficiency (LAL deficiency). LAL deficiency is a genetic disease that leads to liver damage, high blood cholesterol, and other complications due to a build-up of certain types of fats (cholesteryl esters and triglycerides). HOW KANUMA WORKS This medicine is an enzyme replacement therapy. This means that it replaces the missing or defective LAL enzyme in patients with LAL deficiency. This medicine works by lowering the build-up of fat that causes medical complications, including impaired growth, liver damage and heart complications. It also improves blood levels of fats, including elevated LDL (bad cholesterol) and triglycerides. 2. WHAT YOU NEED TO KNOW BEFORE KANUMA IS GIVEN YOU MUST NOT BE GIVEN KANUMA - If you or your child has experienced life-threatening allergic Прочетете целия документ
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 KANUMA 2 mg/ml concentrate for solution for infusion 2. QUALITATIVE AND QUANTITATIVE COMPOSITION Each ml of concentrate contains 2 mg sebelipase alfa*. Each vial of 10 ml contains 20 mg sebelipase alfa. * produced in egg white of transgenic _Gallus_ by recombinant DNA (rDNA) technology. Excipient with known effect Each vial contains 33 mg sodium. For the full list of excipients, see section 6.1. 3. PHARMACEUTICAL FORM Concentrate for solution for infusion (sterile concentrate). Clear to slightly opalescent, colourless to slightly coloured solution. 4. CLINICAL PARTICULARS 4.1 THERAPEUTIC INDICATIONS KANUMA is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) deficiency. 4.2 POSOLOGY AND METHOD OF ADMINISTRATION KANUMA treatment should be supervised by a healthcare professional experienced in the management of patients with LAL deficiency, other metabolic disorders, or chronic liver diseases. KANUMA should be administered by a trained healthcare professional who can manage medical emergencies. Posology It is important to initiate treatment as early as possible after diagnosis of LAL deficiency. For instructions on the preventive measures and monitoring of hypersensitivity reactions, see section 4.4. Following the occurrence of a hypersensitivity reaction, appropriate pre-treatment should be considered according to the standard of care (see section 4.4). 3 _Patients with Rapidly Progressive LAL Deficiency Presenting within the First 6 Months of Life _ The recommended starting dose in infants (< 6 months of age) presenting with rapidly progressive LAL deficiency is either 1 mg/kg or 3 mg/kg administered as an intravenous Прочетете целия документ