Riik: Euroopa Liit
keel: inglise
Allikas: EMA (European Medicines Agency)
alipogene tiparvovec
uniQure biopharma B.V.
C10AX10
alipogene tiparvovec
Lipid modifying agents
Hyperlipoproteinemia Type I
Glybera is indicated for adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD) and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions. The diagnosis of LPLD has to be confirmed by genetic testing. The indication is restricted to patients with detectable levels of LPL protein.
Revision: 8
Withdrawn
2012-10-25
26 B. PACKAGE LEAFLET Medicinal product no longer authorised 27 PACKAGE LEAFLET: INFORMATION FOR THE USER GLYBERA 3 X 10 12 GENOME COPIES /ML SOLUTION FOR INJECTION Alipogene tiparvovec ▼ 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 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 effect not listed in the leaflet. - You have been given a patient card by your doctor. Read it carefully and follow the related instructions. - You should present this card to your health care professionals (doctor, nurse) upon consultation or hospitalisation. See section 4 WHAT IS IN THIS LEAFLET : 1. What Glybera is and what it is used for 2. What you need to know before you are given Glybera 3. How Glybera is given to you 4. Possible side effects 5. How to store Glybera 6. Contents of the pack and other information 1. WHAT GLYBERA IS AND WHAT IT IS USED FOR Glybera contains alipogene tiparvovec, a gene therapy product that works by delivering a gene into the body to correct a genetic deficiency. It belongs to a group of medicines called lipid modifying agents. Glybera is used to treat a specific inherited condition known as “lipoprotein lipase deficiency (LPLD)”. Lipoprotein lipase (LPL) is a naturally occurring substance in the body (known as an enzyme) that controls the level of certain fats in the blood. In lipoprotein lipase deficiency, this enzyme is missing due to a genetic defect. People who are affected by this condition have a build up of very high fat levels in their blood (hyperchylomicronemia). Glybera is used to treat adult patients diagnosed with lipoprotein lipase deficiency (L Lugege kogu dokumenti
1 ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS Medicinal product no longer authorised 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 Glybera 3 × 10 12 genome copies/ml solution for injection 2. QUALITATIVE AND QUANTITATIVE COMPOSITION 2.1 GENERAL DESCRIPTION Alipogene tiparvovec contains the human lipoprotein lipase (LPL) gene variant LPL S447X in a vector. The vector comprises a protein shell derived from adeno-associated virus serotype 1 (AAV1), the Cytomegalovirus (CMV) promoter, a woodchuck hepatitis virus posttranscriptional regulatory element and AAV2 derived inverted terminal repeats. Alipogene tiparvovec is produced using insect cells and recombinant baculovirus technology. 2.2 QUALITATIVE AND QUANTITATIVE COMPOSITION Each vial of alipogene tiparvovec contains 1 extractable ml of solution, containing 3 x 10 12 genome copies (gc). Each patient-specific pack contains a sufficient amount of vials to dose each patient with 1 x 10 12 LPL S447X gc/kg bodyweight. Excipient with known effect: This medicinal product contains 47.5 mg sodium per administration at 27 injection sites to 105.6 mg sodium per administration at 60 injection sites. For the full list of excipients, see section 6.1. 3. PHARMACEUTICAL FORM Solution for injection. Clear, to slightly opalescent, colourless solution. 4. CLINICAL PARTICULARS 4.1 THERAPEUTIC INDICATIONS Glybera is indicated for adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD) and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions . The diagnosis of LPLD has to be confirmed by genetic testing. The indication is restricted to patients with detectable levels of LPL protein (see section 4.4). 4.2 POSOLOGY AND METHOD OF ADMINISTRATION Glybera should only be used when the diagnosis Lugege kogu dokumenti