Source: FDA, National Drug Code (US) Revision Year: 2023
CASGEVY (exagamglogene autotemcel) is a cellular gene therapy consisting of autologous CD34+ HSCs edited by CRISPR/Cas9-technology at the erythroid specific enhancer region of the BCL11A gene to reduce BCL11A expression in erythroid lineage cells, leading to increased fetal hemoglobin (HbF) protein production.
CASGEVY is prepared from the patient’s own HSCs, which are obtained via apheresis procedure(s). The autologous cells are enriched for CD34+ cells, and then genome edited ex vivo by introducing the CRISPR/Cas9 ribonucleoprotein (RNP) complex by electroporation. The guide RNA included in the RNP complex enables CRISPR/Cas9 to make a precise DNA double-strand break at a 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 disrupted and BCL11A expression is reduced. This reduction in BCL11A expression conversely results in an increase in gamma-globin expression and downstream fetal hemoglobin formation.
The edited CD34+ cells are formulated into a suspension in a sterile cryo-preservative medium and cryopreserved. CASGEVY is shipped as a frozen suspension in patient-specific vial(s). The product is thawed prior to infusion, and administered as a HSC transplant [see Dosage and Administration (2.2), How Supplied/Storage and Handling (16)]. Due to the presence of cells, the thawed product may be clear to slightly cloudy and may contain small inherent proteinaceous particles or visible cell aggregates.
The formulation contains 5% dimethyl sulfoxide (DMSO) and dextran 40.
| Dosage Forms and Strengths |
|---|
|
CASGEVY is a cell suspension for intravenous infusion. A single dose of CASGEVY is composed of one or more vials. Each vial contains 4 to 13 × 106 CD34 cells/mL suspended in 1.5 to 20 mL cryopreservative medium [see How Supplied/Storage and Handling (16)]. The minimum recommended dose of CASGEVY is 3 × 106 CD34 cells per kg of body weight. See the Lot Information Sheet for actual strength and dose. The Lot Information Sheet is included inside the lid of the liquid nitrogen dry shipper used to transport CASGEVY. |
| How Supplied |
|---|
|
CASGEVY is supplied in one or more vials containing a frozen suspension of genome edited autologous CD34+ cells in a cryo-preservative medium containing 5% DMSO and dextran 40. NDC 51167-290-09 CASGEVY is stored in the vapor phase of liquid nitrogen and is shipped from the manufacturing facility to the treatment center storage facility in a cryoshipper. CASGEVY is supplied in vial(s) packaged in carton(s). One carton contains a single lot of CASGEVY consisting of 1 to 9 vials. A single dose of CASGEVY may consist of multiple CASGEVY lots, and therefore may consist of multiple cartons. A Lot Information Sheet listing the total dose of CASGEVY is affixed inside the shipper. Manufactured for: Vertex Pharmaceuticals Incorporated, Boston, MA 02210 |
| 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.
