البلد: الاتحاد الأوروبي
اللغة: الإنجليزية
المصدر: EMA (European Medicines Agency)
elivaldogene autotemcel
bluebird bio (Netherlands) B.V.
N07
elivaldogene autotemcel
Other nervous system drugs
Adrenoleukodystrophy
Treatment of early cerebral adrenoleukodystrophy in patients less than 18 years of age, with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA) matched sibling haematopoietic stem cell donor is not available.
Withdrawn
2021-07-16
35 B. PACKAGE LEAFLET Medicinal product no longer authorised 36 PACKAGE LEAFLET: INFORMATION FOR THE PATIENT OR CARER SKYSONA 2-30 × 10 6 CELLS/ML DISPERSION FOR INFUSION elivaldogene autotemcel (autologous CD34 + cells encoding _ABCD1_ gene) 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/YOUR CHILD IS 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 the doctor or nurse. - If you get any side effects, talk to the doctor or nurse. This includes any possible side effects not listed in this leaflet. See section 4. - The doctor or nurse will give you a Patient Alert Card which contains important safety information about the treatment with Skysona. Read it carefully and follow the instructions on it. - Carry the Patient Alert Card with you at all times and always show it to any doctor or nurse who sees you or if you go to the hospital. WHAT IS IN THIS LEAFLET 1. What Skysona is and what it is used for 2. What you need to know before you or your child is given Skysona 3. How Skysona is made and given 4. Possible side effects 5. How to store Skysona 6. Contents of the pack and other information 1. WHAT SKYSONA IS AND WHAT IT IS USED FOR Skysona is used to treat a serious genetic disease called cerebral adrenoleukodystrophy (CALD) in children and adolescents younger than 18 years old. People with CALD have an alteration in the gene that makes a protein called adrenoleukodystrophy protein (ALDP). People with CALD either cannot make this protein or have a protein that does not work well. This leads to the build-up of very long chain fatty acids in the body, particularly in the brain. These fatty acids cause severe damage to brain cells. If untreated, this damage will lead اقرأ الوثيقة كاملة
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 Skysona 2-30 × 10 6 cells/mL dispersion for infusion 2. QUALITATIVE AND QUANTITATIVE COMPOSITION 2.1 GENERAL DESCRIPTION Skysona (elivaldogene autotemcel) is a genetically modified autologous CD34 + cell-enriched population that contains haematopoietic stem cells (HSCs) transduced _ex vivo_ with lentiviral vector (LVV) encoding _ABCD1 _ complementary deoxyribonucleic acid (cDNA) for human adrenoleukodystrophy protein (ALDP). 2.2 QUALITATIVE AND QUANTITATIVE COMPOSITION Each patient-specific infusion bag of Skysona contains elivaldogene autotemcel at a batch-dependent concentration of genetically modified autologous CD34 + cell enriched population. The finished product is packaged in one or more infusion bags, containing a dispersion of 2-30 × 10 6 cells/mL of CD34 + enriched cell population suspended in cryopreservative solution. Each infusion bag contains approximately 20 mL of dispersion for infusion. The quantitative information of medicinal product regarding strength, CD34 + cells, and dose for the specific patient is provided in the Lot Information Sheet. The Lot Information Sheet is included inside the lid of the cryoshipper used to transport Skysona. Excipient(s) with known effect Each dose contains 391-1564 mg sodium (included in Cryostor CS5). For the full list of excipients, see section 6.1. 3. PHARMACEUTICAL FORM Dispersion for infusion. A colourless to white to red, including shades of white or pink, light yellow, and orange dispersion. Medicinal product no longer authorised 3 4. CLINICAL PARTICULARS 4.1 THERAPEUTIC INDICATIONS Skysona is indicated for the treatment of early cerebral adrenoleukodystrophy in patients less than 18 yea اقرأ الوثيقة كاملة