Source: European Medicines Agency (EU) Revision Year: 2024 Publisher: Vertex Pharmaceuticals (Ireland) Limited, Unit 49, Block F2, Northwood Court, Santry, Dublin 9, D09 T665, Ireland
Casgevy 4-13 × 106 cells/mL dispersion for infusion.
| Pharmaceutical Form |
|---|
|
Dispersion for infusion. A translucent cell dispersion for infusion, free from foreign particles. |
Casgevy (exagamglogene autotemcel) is a genetically modified autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPCs) edited ex vivo by CRISPR/Cas9 at the erythroid-specific enhancer region of the BCL11A gene.
Each patient-specific vial of Casgevy contains exagamglogene autotemcel at a batch-dependent concentration of genetically modified autologous CD34+ cells enriched population. The medicinal product is packaged in one or more vials overall containing a dispersion for infusion of 4-13 × 106 cells/mL of viable CD34+ cells enriched population suspended in a cryopreservative solution.
Each vial contains 1.5 to 20 mL of dispersion for infusion.
The quantitative information of medicinal product, including the number of vials (see section 6) to be administered, is presented in the Lot information sheet (LIS) located inside the lid of the cryoshipper used for transport.
Excipient(s) with known effect:
This medicinal product contains 50 mg of dimethyl sulfoxide (DMSO) per mL.
This medicinal product contains 3.5 mg of sodium per mL.
For the full list of excipients, see section 6.1.
| Active Ingredient | Description | |
|---|---|---|
| Exagamglogene autotemcel |
Exagamglogene autotemcel is a cell therapy consisting of autologous CD34+ HSPCs ex vivo edited by CRISPR/Cas9-technology. The highly specific guide RNA enables CRISPR/Cas9 to make a precise DNA double-strand break at the critical transcription factor binding site (GATA1) in the erythroid specific enhancer region of the BCL11A gene. As a result of the editing, GATA1 binding is irreversibly disrupted and BCL11A expression reduced. Reduced BCL11A expression results in an increase in γ-globin expression and foetal haemoglobin (HbF) protein production in erythroid cells, addressing the absent globin in transfusion-dependent β-thalassemia (TDT) and the aberrant globin in sickle cell disease (SCD), which are the underlying causes of disease. |
| List of Excipients |
|---|
|
CryoStor CS5 (contains dimethyl sulfoxide and dextran 40) |
Casgevy is supplied in cryopreservation vials made of cyclic olefin copolymer. Each vial contains 1.5 ml to 20 ml of Casgevy.
Vials are packed in a paperboard carton box. Each carton may contain up to 9 vials. The final outer carton contains a variable number of vials according to the patient specific dose required.
Casgevy is shipped from the manufacturing facility to the treatment centre storage facility in a cryoshipper. One cryoshipper may contain multiple cartons, which may contain multiple vials, all intended for a single patient.
Vertex Pharmaceuticals (Ireland) Limited, Unit 49, Block F2, Northwood Court, Santry, Dublin 9, D09 T665, Ireland
EU/1/23/1787/001
| Drug | Countries | |
|---|---|---|
| CASGEVY | Croatia, Lithuania, United Kingdom, United States |
© All content on this website, including data entry, data processing, decision support tools, "RxReasoner" logo and graphics, is the intellectual property of RxReasoner and is protected by copyright laws. Unauthorized reproduction or distribution of any part of this content without explicit written permission from RxReasoner is strictly prohibited. Any third-party content used on this site is acknowledged and utilized under fair use principles.
