Source: European Medicines Agency (EU) Revision Year: 2021 Publisher: bluebird bio (Netherlands) B.V., Stadsplateau 7, WTC Utrecht, 3521AZ Utrecht, The Netherlands
Skysona 2-30 × 106 cells/mL dispersion for infusion.
| Pharmaceutical Form |
|---|
|
Dispersion for infusion. A colourless to white to red, including shades of white or pink, light yellow, and orange dispersion. |
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).
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 × 106 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.
| Active Ingredient | Description | |
|---|---|---|
| Elivaldogene autotemcel |
Elivaldogene autotemcel is indicated for the treatment of early cerebral adrenoleukodystrophy in patients less than 18 years of age, with an ABCD1 genetic mutation. Elivaldogene autotemcel adds functional copies of the ABCD1 cDNA into patients' HSCs through transduction of autologous CD34+ cells with Lenti-D LVV. Following successful engraftment with genetically modified cells, the expression of ALDP is expected to be life-long. |
| List of Excipients |
|---|
|
Cryostor CS5 |
Approximately 20 mL in a fluoro ethylenepropylene infusion bag(s), each packed in a transparent pouch inside a metal cassette.
Skysona is shipped from the manufacturing facility to the infusion centre storage facility in a cryoshipper, which may contain multiple metal cassettes intended for a single patient. Each metal cassette contains one infusion bag with Skysona. One lot of drug product may be packaged in either one or two 20 mL bags, depending on the total number of cells present. Multiple lots may be administered to the patient as a single dose.
bluebird bio (Netherlands) B.V., Stadsplateau 7, WTC Utrecht, 3521AZ Utrecht, The Netherlands
EU/1/21/1563/001
| Drug | Countries | |
|---|---|---|
| SKYSONA | Estonia, France, Lithuania, Poland |
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